News

SAR 302503 (Sanofi) successs in JAKARTA study of Myelofibrosis

Sanofi has announced that the pivotal study, JAKARTA, examining the selective JAK2 inhibitor SAR 302503 for Myelofibrosis versus placebo in 289 patients, met its primary endpoint in both dose groups. The primary endpoint assessed the proportion of patients achieving >35% reduction of spleen volume. Consistent with data reported in previous trials, the most common adverse events were anemia, diarrhea, ...

Phase III trial for N9-GP(Novo Nordisk) treatment for Haemophilia B

Novo Nordisk has announced the completion of PARADIGM 2, the first Phase III trial with a long-acting FIX derivative, N9-GP (glycopegylated recombinant factor IX), for Haemophilia B patients. PARADIGM 2 is a multi-centre, blinded trial evaluating the safety and efficacy of N9-GP when used for on demand or prophylactic treatment in patients with Haemophilia B.

In the trial, ...

Moxetumomab (MedImmune/AstraZeneca) moves into Phase III for Treatment of Hairy Cell Leukemia

MedImmune, AstraZeneca’s global biologics research and development arm, has enrolled the first patient in the Phase III clinical trial for moxetumomab pasudotox. The trial is sponsored by the Cancer Therapy Evaluation Program (CTEP), a programme within the Division of Cancer Treatment and Diagnosis at the US National Cancer Institute, and will evaluate moxetumomab pasudotox as a potential treatment in ...

Seattle Genetics files Adcetris at FDA for extended use in Hodgkin Lymphoma and sALCL

Seattle Genetics, Inc. has announced that the FDA has accepted for filing a supplement to the Biologics License Application (sBLA) supporting the use of Adcetris (brentuximab vedotin) for retreatment and extended duration beyond 16 cycles of therapy in relapsed Hodgkin Lymphoma (HL) and Systemic Anaplastic Large Cell Lymphoma (sALCL). The FDA is expected to take action on the application ...

FDA accepts BLA from Biogen for Eloctate for treatment of Haemophilia A

Biogen Idec has announced that the FDA has accepted the company's Biologics License Application (BLA) for the marketing approval of Eloctate (recombinant factor VIII Fc fusion protein) for the treatment of Haemophilia A. Eloctate is the first Haemophilia A product candidate in a new class of long-lasting clotting factor therapies being developed with the goal of providing long-lasting protection and ...

FDA approves Nymalize (Arbor Pharmaceuticals) for Sub Arachnoid Haemorrhage

The FDA has approved the New Drug Application (NDA) for Nymalize (nimodipine) oral solution, from Arbor Pharmaceuticals, for the improvement of neurological outcome by reducing the incidence and severity of ischemic deficits in adult patients with Sub Arachnoid Haemorrhage. It is the only nimodipine oral solution indicated for the improvement of neurological outcome in adult patients with SAH. The company ...

Enzastaurin (Eli Lilly) fails Phase III trial for B Cell Lymphoma

Eli Lilly and Company has announced Phase III clinical trial results from enzastaurin's PRELUDE study, which explored the molecule as a monotherapy in the prevention of relapse in patients with diffuse large B-cell lymphoma (DLBCL). The study failed to show a statistically significant increase compared to placebo in disease-free survival in patients at high risk of relapse following rituximab-based ...

FDA approves Liptruzet (Merck Inc) for Dyslipidaemia

The FDA has approved Liptruzet (ezetimibe and atorvastatin) tablets, from Merck Inc, for the treatment of Dyslipidaemia. It is indicated for elevated low-density lipoprotein (LDL) cholesterol in patients with primary or mixed hyperlipidemia as adjunctive therapy to diet when diet alone is not enough. Approval is based on results including a multicenter, clinical study in which 628 patients with hyperlipidemia ...

CHMP recommends Revlimid for treatment of Myelodysplastic Syndromes

The Committee for Medicinal Products for Human Use (CHMP) has recommended Revlimid (lenalidomide)from Celgene for the treatment of patients with transfusion-dependent anaemia due to low- or intermediate-1-risk Myelodysplastic Syndromes associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate.

FDA approves Kcentra (CSL Behring)to reverse vitamin K antagonist anticoagulation with major bleeding

The FDA on 29 April 2013 has approved Kcentra (Prothrombin Complex Concentrate, Human) for the urgent reversal of vitamin K antagonist (VKA) anticoagulation in adults with acute major bleeding. Plasma is the only other product approved for this use in the United States. Patients receiving chronic anticoagulation therapy with warfarin and other VKA anticoagulants to prevent blood clotting in conditions ...

NICE confirms rejection of Jakavi(Novartis) for Myelofibrosis

NICE has confirmed that it will not recommend Jakavi(ruxolitinib) from Novartis after it found problems with its data and the costing model. NICE has published final draft guidance not recommending Jakavi for the treatment of disease-related splenomegaly (enlarged spleen) or its symptoms in adults with primary Myelofibrosis, or post polycythaemia vera Myelofibrosis or post essential thrombocythaemia Myelofibrosis.

Novartis ...

EU extends Berinert (CSL Behring) indication for pre-procedure prevention of acute HAE episodes

The EU has approved an extended use of Berinert (C1 inhibitor concentrate), from CSL Behring, for pre-procedure prevention (short-term prophylaxis) of acute episodes of Hereditary Angio-Oedema (HAE) in adult and pediatric patients undergoing medical, dental or surgical procedures. Berinert is already indicated in Europe for the treatment of acute attacks of HAE at all body sites in adults and children. ...

NICE rejects Pixuvri (Cell Therapeutics) for the treatment of Non-Hodgkin's Lymphoma

NICE has issued draft guidance not recommending the use of Pixuvri (pixantrone), from Cell Therapeutics, for an aggressive form of Non-Hodgkin's Lymphoma. The committee concluded there was insufficient evidence to show that the drug is more effective than current treatments and also found that it does not represent good value for money. According to NICE, the clinical trial evidence submitted ...

Phase II data shows PCI 32765 (Pharmacyclics Inc.) provides sustained control in CLL patients

Results from a Phase II trial of PCI 32765 (ibrutinib), from Pharmacyclics Inc., demonstrates the drug offers rapid and sustained control as a monotherapy in untreated, relapsed and refractory Chronic Lymphocytic Leukemia (CLL) patients, irrespective of characteristics that predict poor outcomes to chemoimmunotherapy. It included an analysis of two CLL patient cohorts: the elderly, above 65 years of age, (n=24) ...

Elacyt (Clavis Pharma) fails Phase III trial for AML

Clavis Pharma announced results of the Phase III CLAVELA study investigating Elacyt (elacytarabine) in patients with relapsed or refractory Acute Myeloid Leukaemia (AML). The trial showed that there was no significant difference in overall survival between the two arms where patients were randomised to receive either elacytarabine or investigator's choice of treatment. Median survival in the elacytarabine arm was ...

European Commission grants conditional marketing approval to Bosulif (Pfizer) for PH+CML

The European Commission (EC) has granted conditional marketing authorization for Bosulif (bosutinib) from Pfizer, in the European Union for the treatment of adult patients with chronic phase (CP), accelerated phase (AP) and blast phase (BP) Philadelphia chromosome positive Chronic Myelogenous Leukemia (Ph+ CML) previously treated with one or more tyrosine kinase inhibitor(s) and for whom imatinib, nilotinib and dasatinib ...

CHMP recomends Iclusig (Ariad Pharma) for CML and Ph+ALL

Ariad Pharmaceuticals, Inc. has announced that the Committee for Human Medicinal Products (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion on the marketing authorization application for Iclusig (ponatinib) for two indications: • The treatment of adult patients with chronic phase, accelerated phase or blast phase Chronic Myeloid Leukaemia (CML) who are resistant to dasatinib or nilotinib; ...

Phase III trial of Aranesp (Amgen) in anemic Heart Failure patients shows drug offers no real benefit

Results from the RED-HF trial involved 2,278 anemic Heart Failure patients given either Aranesp (darbepoetin alfa), from Amgen, or placebo shows the drug does not improve patients' health, nor does it reduce their risk of death from Heart Failure. In the darbepoetin alfa group, 50.7 percent of the patients experienced death from any cause or hospitalization for worsening Heart Failure. ...

Phase III study of perifosine (Aeterna Zentaris )for Multiple Myeloma discontinued

Aeterna Zentaris Inc. has announced that an independent Data Safety Monitoring Board ("DSMB") has recommended discontinuing the ongoing Phase III study comparing the efficacy and safety of perifosine to placebo when combined with bortezomib (Velcade) and dexamethasone in patients with relapsed or relapsed/refractory Multiple Myeloma. Based on the outcome of its pre planned interim analysis of efficacy and safety, ...

Biogen IDEC files rFVIII Fc at FDA for treatment of Haemophilia A

Biogen Idec has announced that the company has submitted a Biologics License Application (BLA) to the FDA for the marketing approval of recombinant factor VIII Fc fusion protein (rFVIIIFc) for the treatment of Haemophilia A. Recombinant FVIIIFc is the first Haemophilia A product candidate in a new class of long-lasting clotting factor therapies being developed with the goal of ...

Tredaptive (Merck Inc) fails to meet primary end point in HPS2-THRIVE Dyslipidaemia trial

Detailed results of HPS2-THRIVE, a study of Tredaptive (nicotinic acid plus laropiprant), from Merck Inc, in 25673 patients with Dyslipidaemia suggests adding Tredaptive to statin therapy did not significantly further reduce the risk of major vascular events. In the trial the composite primary endpoint of major vascular events (coronary death non-fatal heart attack stroke or any arterial revascularization) occurred in ...

Phase IV study of Livalo (Eli Lilly/Kowa Pharmaceuticals) meets end point in patients with Dyslipidemia

Results of a Phase IV study evaluating the efficacy of Livalo (pitavastatin), from Eli Lilly/Kowa Pharmaceuticals, compared with pravastatin in reducing low-density lipoprotein cholesterol (LDL-C) in HIV-infected adults with Dyslipidemia has met its end point. Results showed that, after 12 weeks of therapy, pitavastatin had a significantly greater decrease in LDL-C compared with pravastatin (pitavastatin -49.4 mg/dL and pravastatin -33.6 ...

Results from Phase II study of CHR 2797 (Cell Therapeutics) for AML published in the Lancet Oncology

Results from the OPAL Phase II study of CHR 2797 (tosedostat), from Cell Therapeutics, in elderly patients with relapsed or refractory Acute Myeloid Leukemia (AML) have been published in the Lancet Oncology. Results were released in 2012. The trial showed that once-daily oral tosedostat resulted in a disease control rate of 51%. Subset analyses suggested the greatest benefit occurred in ...

FDA expands approval of Corifact (CSL Behring) to manage surgical bleeding in adult and pediatric patients with Factor XIII Deficiency

The FDA has approved an expansion of the indication for Corifact (factor XIII Concentrate), from CSL Behring, to include the peri-operative management of surgical bleeding in adult and pediatric patients with congenital Factor XIII Deficiency. In 2011, Corifact became the first and only FXIII concentrate approved in the US for the routine prophylactic treatment of congenital factor XIII deficiency. The ...

FDA recall of Omontys (Affymax/Takeda) for treatment of Anaemia in adult dialysis patients

The FDA is alerting health care providers and patients of a voluntary nationwide recall of all lots of Omontys Injection (peginesatide) by Affymax, Inc., and Takeda Pharmaceuticals Company Limited. The recall is due to reports of anaphylaxis, a serious and life-threatening allergic reaction. Omontys is used to treat anemia in adult dialysis patients.

Omontys was filed last year at ...

NICE does not recommend Jakavi (Novartis) for Myelofibrosis

NICE (National Institute for Health and Clinical Excellence) in its draft guidance stated that it is not recommending Jakavi (ruxolitinib) from Novartis, for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adults with primary Myelofibrosis, or Myelofibrosis secondary to polycythaemia vera or essential Thrombocythaemia.

NICE stated that although the drug might offer a survival benefit, ...

PA 21( Galencia) is filed at FDA and EMA for treatment of Hyperphosphataemia in CKD patients

A New Drug Application (NDA) for PA21 from Fresenius Kabi/Galencia has been submitted to the FDA. Separately, the European Medicines Agency has accepted the Marketing Authorisation Application which was submitted December 2012 to seek approval in the European Union. Further submissions for approval are being prepared.

PA21 is a chewable, iron-based phosphate binder for the control of Hyperphosphatemia in ...

Successful studies for rFVIIIFc and rFIXFc (Biogen/Swedish Orphan) for Haemopilia

Biogen Idec and Swedish Orphan Biovitrum (Sobi)released data that confirmed the ability of investigational recombinant factors VIII Fc fusion protein (rFVIIIFc) and IX Fc fusion protein (rFIXFc) to provide long-lasting protection from bleeding with fewer injections than are required with the current standard of care for people with Haemophilia. The data, from the largest phase III registrational studies conducted ...

FDA grants accelerated approval to Pomalyst (Celgene) to treat Multiple Myeloma

The FDA on 8 February 2013, approved Pomalyst (pomalidomide), from Celgene, to treat patients with Multiple Myeloma whose disease progressed after being treated with other cancer drugs. Pomalyst is a pill that modulates the body’s immune system to destroy cancerous cells and inhibit their growth. It is intended for patients who have received at least two prior therapies, including ...

Soluble Ferric Pyrophosphate (Rockwell Medical) success in Phase III trial for Iron Deficiency

Rockwell Medical announced successful topline results from the PRIME clinical study of Soluble Ferric Pyrophosphate (SFP), its investigational iron-delivery drug currently in Phase III clinical studies for the treatment of Iron Deficiency in hemodialysis patients. The PRIME study demonstrated that regular administration of SFP-iron via dialysate reduced the usage of erythropoietin stimulating agents (ESAs) during hemodialysis by 37.1% while ...

Obinutuzumab (Genentech) success in Phase III trial for CLL

Genentech has announced positive results from Stage 1 of CLL11, a Phase III randomized study to investigate the efficacy and safety profile of the investigational medicine obinutuzumab (GA101) plus chlorambucil chemotherapy compared with chlorambucil alone in people with previously untreated Chronic Lymphocytic Leukemia (CLL). An improvement in progression-free survival (PFS) was achieved as GA101 plus chlorambucil significantly reduced the risk ...

FDA approves Kynamro (Genzyme/Sanofi) to treat Homozygous Familial Hypercholesterolemia

The FDA 0n 29 January 2013 approved Kynamro (mipomersen sodium) injection, from Genzyme/Sanofi, as an addition to lipid-lowering medications and diet to treat patients with a rare type of high cholesterol called Homozygous Familial Hypercholesterolemia (HoFH). The addition of Kynamro helps to reduce low-density lipoprotein-cholesterol (LDL-C), apolipoprotein B, total cholesterol, and non-high density lipoprotein-cholesterol (non HDL-C).

HoFH, an ...

Zerenex (Keryc Biophrama) succuss in Phase III trial for Hyperphosphatemia

Keryx Biopharmaceuticals, Inc.announced successful top-line results from the long-term Phase III study of Zerenex (ferric citrate), the Company's ferric iron-based phosphate binder drug candidate, for the treatment of elevated serum phosphorus levels, or Hyperphosphatemia, in patients with end-stage renal disease (ESRD) on dialysis. In this study, Zerenex met the study's primary endpoint, described below, demonstrating a highly statistically significant change ...

Phase III study of Neulasta (Amgen) for Neutropenia in Colorectal Cancer meets primary endpoint

PAVES, a Phase III trial evaluating Neulasta (pegfilgrastim), from Amgen, in 845 patients receiving FOLFOX or FOLFIRI and bevacizumab for first-line treatment of metastatic Colorectal Cancer, has met its primary endpoint, with Neulasta significantly reducing the incidence of febrile Neutropenia. All patients received treatment with either FOLFOX or FOLFIRI plus bevacizumab and were randomized to receive either placebo or 6 ...

FDA approves Gleevec (Novartis) to treat Ph+ALL

The FDA on 25 January 2013 approved a new use of Gleevec (imatinib) to treat children newly diagnosed with Philadelphia chromosome positive (Ph+) acute lymphoblastic leukemia (ALL). ALL is the most common type of pediatric cancer, affecting approximately 2,900 children annually, and progresses quickly if untreated. Children with Ph+ ALL have a genetic abnormality that causes proteins called tyrosine kinases ...

Soliris (Alexion Pharma) rejected by UK Health Ministers for Haemolytic Uraemic Syndrome

Soliris (eculizumab)from Alexion Pharma was referred in the UK to the Advisory Group for National Specialised Services (AGNSS) instead of NICE and AGNSS recommended the $400 000 per patient, per year, drug for treatment of patients with atypical Haemolytic Uraemic Syndrome (aHUS), a rare, chronic blood disease. The decision of AGNSS was overruled by Health Ministers and the application has ...

FDA approves Exjade (Novartis) to treat Non-Transfusion-Dependent Thalassemia

The FDA on 23 January 2013 expanded the approved use of Exjade (deferasirox) from Novartis, to treat patients ages 10 years and older who have chronic iron overload resulting from a genetic blood disorder called Non-Transfusion-Dependent Thalassemia (NTDT).

NTDT is a milder form of thalassemia that does not require individuals to get frequent red blood cell transfusions. However, over ...

FDA approves octaplas LG (Octapharma) coagulation factor

The FDA has on 18 January 2013, approved octaplas,from Octapharma, a pooled plasma (human) blood product for the replacement of clotting proteins (coagulation factors) in certain medical conditions where patients have insufficient levels. Clotting protein deficiencies can cause excessive bleeding or excessive clotting.

Octaplas has been used extensively in Europe and other countries. A previous generation of octaplas was ...

FEIBA NF success in Phase III trial for Hemophilia A and B

Baxter International Inc. has announced pivotal Phase III study results evaluating the efficacy and safety of routine prophylaxis compared to on-demand treatment of FEIBA NF [Anti-Inhibitor Coagulant Complex], Nanofiltered and Vapor Heated, in patients with Hemophilia A or B and inhibitors

Top-line results from the study showed a reduced median annual bleed rate (ABR) from 28.7 during FEIBA NF ...

BAX 855 enters Phase II/III study for Haemophilia

Baxter International Inc. has announced it has submitted an Investigational New Drug (IND) application for its investigational Haemophilia A treatment, BAX 855, with the FDA following positive results from a Phase I trial. BAX 855 is a full-length longer-acting recombinant factor VIII (rFVIII) that was developed to increase the half-life of Advate [Antihemophilic Factor (Recombinant) Plasma/Albumin-Free Method] - the most ...

Biogen Idec files rFIXFc at FDA for treatment of Haemophilia B

Biogen Idec has submitted a Biologics License Application (BLA) to the FDA for the marketing approval of recombinant factor IX Fc fusion protein (rFIXFc) for the treatment of Haemophilia B.

rFIXFc is the first product candidate in a new class of long-lasting clotting factor therapies that are being developed with the goals of reducing the burden of treatment ...

FDA approves Juxtapid (Aegerion Pharma) for patients with Homozygous Familial Hypercholesterolemia

The FDA has approved Juxtapid (lomitapide), from Aegerion Pharma, as an adjunct to diet and other lipid-lowering treatments to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B) and non-high-density-lipoprotein cholesterol (non-HDL) in patients with Homozygous Familial Hypercholesterolemia (HoFH). The FDA based its approval on a pivotal Phase III study, which evaluated the safety and effectiveness of ...

Tredaptive (Merck Inc) fails in HPS2-THRIVE trial

The HPS2-THRIVE (Heart Protection Study 2-Treatment of HDL to Reduce the Incidence of Vascular Events) study of Tredaptive(extended-release niacin/laropiprant) did not meet its primary endpoint. Merck Inc., and the investigators are informing regulatory agencies of these results. The company is also preparing communications to health care providers in countries where the medicine is currently available, and will continue to ...

EU Commission approves Exjade (Novartis) for treatment of Iron Overload with Thalassemia Syndromes

Novartis announced that the European Commission has approved Exjade (deferasirox) for the treatment of chronic iron overload requiring chelation therapy when deferoxamine therapy is contraindicated or inadequate in patients aged 10 years and older with non-transfusion-dependent thalassemia (NTDT) syndromes. Exjade is the first oral treatment approved in the European Union (EU) specifically indicated for the treatment of chronic iron ...

Phase II study of RiaSTAP (CSL Behring) shows the drug has benefits in Critical Care patients

Clinical study results show that RiaSTAP (fibrinogen concentrate [human]), from CSL Behring, can significantly reduce the need for blood transfusion when given in a Critical Care situation, as an intra-operative, targeted first-line hemostatic therapy in bleeding patients undergoing aortic replacement surgery. The Phase II prospective study, led by Niels Rahe-Meyer of the Clinic for Anesthesiology and Intensive Care Medicine, at ...

Positive results in Phase III trial of Jakafi (Incyte Corporation/Novartis) for patients with Myelofibrosis

New results from the Phase III COMFORT-II study shows that Jakafi(ruxolitinib), from Incyte Corporation, reduces splenomegaly and other symptoms commonly associated with Myelofibrosis. In addition, benefits are maintained for at least two years.

In a follow-up of 112 weeks, of patients randomised to receive either oral ruxolitinib or conventional therapy, just under half of patients receiving ruxolitinib achieved better than ...

NICE recommends Revolade (GSK) for ITP

The National Institute for Health and Clinical Excellence(NICE) now recommends the use of Revolade (eltrombopag olamine)for patients with the bleeding disorder Chronic Immune Thrombocytopenic Purpura (ITP). The recommendation is for use of the drug for treating ITP in patients who have had their spleens removed or those refractory to other treatments, or as a second line treatment in those who ...

FDA approves Signifor (Novartis) for treatment of Cushings Disease

The FDA has approved Signifor (pasireotide) injection from Novartis, for the treatment of adult patients with Cushing's Disease for whom pituitary surgery is not an option or has not been curative. Signifor is the first medicine to be approved in the US that addresses the underlying mechanism of Cushing's Disease, a serious, debilitating endocrine disorder caused by the presence ...

Positive results in Phase II study of BI 6727 (Boehringer) in patients with AML

Phase II results from an interim analysis of a study involving BI 6727 (volasertib), from Boehringer, in newly diagnosed patients with Acute Myeloid Leukemia (AML) shows higher rates of objective response and an improvement in event free survival in patients treated with volasertib in combination with low-dose cytarabine (LDAC), compared to patients treated with LDAC alone. Objective responses were observed ...

CHMP rejects Kynamro (Sanofi) for treatment of Homozygous Familial Hypercholesterolemia

Genzyme, a Sanofi company and Isis Pharmaceuticals Inc. have announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a negative opinion for its marketing authorization application for Kynamro (mipomersen) for the treatment of patients with Homozygous Familial Hypercholesterolaemia (HoFH). Genzyme plans to request a re-examination of the CHMP Opinion....

Study shows benefits for bone growth in infants fed on InFat (Advanced Lipids)

A randomized, double-blind, controlled clinical trial on bone strength in term infants fed InFat, a high beta-palmitate formula, from Advanced Lipids, indicates InFat leads to significantly higher bone-strength parameters compared to a standard vegetable oil control. The trial studied the effect of 12 weeks feeding of infant formula with InFat on bone strength parameters. Bone strength results were measured by ...

Phase II study success of quizartinib (Astellas/Ambit Bioscience) in AML

Astellas Pharma Inc. and Ambit Biosciences Corporation announced the results from a completed Phase II study with the investigational FLT3 inhibitor, quizartinib (AC220), as an oral monotherapy treatment regimen in patients with relapsed or refractory Acute Myeloid Leukemia (AML). Data from two study cohorts (patients aged ? 60 years with AML relapsed in <1 year or refractory to 1st-line chemotherapy; ...

The Medicines Company and BMS collaborate in marketing Recothrom for Haemostasis.

The Medicines Company and Bristol-Myers Squibb Company announced that the companies have signed a global license and two year collaboration for Recothrom, a recombinant thrombin approved by the FDA for use as a topical hemostat to control non-arterial bleeding during surgical procedures.Recothrom was approved by the FDA in January 2008 and is marketed in Canada by Bayer HealthCare....

Two Phase III trials show benefits of Tasigna (Novartis) for patients with Ph+ CML

New results from two Phase III clinical trials further establish the benefits of Tasigna (nilotinib), from Novartis, compared to Gleevec (imatinib mesylate) in the treatment of Philadelphia chromosome-positive Chronic Myeloid Leukemia (Ph+ CML) in newly diagnosed patients and in those with residual disease who switched to Tasigna after long-term treatment with Gleevec. Two-year results from ENESTcmr showed that switching to ...

Phase III results for BAX 326 (Baxter) for Haemophilia B

A Phase I/III prospective, controlled, multicenter study investigated the pharmacokinetics, efficacy and safety of BAX 326 from Baxter, in 73 previously-treated patients with severe or moderately severe Hemophilia B.

Results from the study showed that twice-weekly prophylactic treatment with BAX 326 achieved a median annualized bleed rate of 1.99 with 43 percent of patients experiencing no bleeds. The ...

Phase III trial success for PCI 32765 (Janssen Biotech) in CLL treatment

Research demonstrates that PCI 32765 (ibrutinib) from Pharmacyclics/Janssen Biotech, may be an effective and safe targeted treatment option for previously untreated, hard-to-treat, and relapsed patients with Chronic Lymphocytic Leukemia (CLL).

A study enrolled 116 CLL patient participants in several treatment cohorts: patients who were never treated (the treatment-naive group), those who had received two or more prior therapies ...

Roche submits extension application to EMA for subcutaneous MabThera

Roche has submitted a line extension application to the European Medicines Agency for a subcutaneous (SC) formulation of MabThera (rituxumab) for patients with Non-Hodgkin Lymphoma. Currently, MabThera is available as an IV formulation. The application is backed by pivotal study data which showed that SC administration of MabThera enabled the delivery of MabThera over approximately five minutes without compromising its ...

FDA approves Evarrest (Ethicon Biosurgery) for problematic bleeding during Surgery

The FDA has approved Evarrest fibrin sealant patch, from Ethicon Biosurgery, a product that rapidly aids in stopping problematic bleeding during Surgery. Evarrest consists of a coating of biologics and a flexible patch that work together to encourage the hemostasis process. Clinical studies demonstrate that Evarrest is 98% effective in stopping bleeding and maintaining hemostasis compared to the current standard ...

Orphan Europe acquires European rights to GRASPA for ALL

Recordati announces that its subsidiary Orphan Europe and Erytech Pharma, a late development stage French biopharmaceutical company focused on orphan oncology and rare diseases, have entered into an agreement granting Orphan Europe the exclusive rights for the commercialization and distribution of Graspa for the treatment of Acute Lymphoblastic Leukemia (ALL) and Acute Myeloid Leukemia (AML) in Europe. Graspa, (human erythrocytes ...

FDA approves Signifor (Novartis) for treatment of Cushing's Disease

The FDA Endocrinologic and Metabolic Drugs Advisory Committee has voted unanimously in support of the use of Signifor (pasireotide) from Novartis, for the treatment of patients with Cushing's Disease who require medical therapeutic intervention. The recommendation was based on data from clinical trials of pasireotide, including PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio - CUSHING'S disease), the largest randomized Phase III study ...

Positive results in Phase III study of lomitapide (Aegerion Pharmaceuticals) for Homozygous Familial Hypercholesterolemia

New results from a Phase III clinical trial of lomitapide, from Aegerion Pharmaceuticals, show it substantially and stably reduced LDL cholesterol in patients with the orphan disease Homozygous Familial Hypercholesterolemia (HoFH). Twenty-nine adult HoFH patients from across the world were enrolled, with 23 patients completing both the efficacy and the safety phases. All of the patients received lomitapide along with ...

AMG 134 success in GAUSS study in patients intolerant of statins

Amgen has announced positive results from the AMG 145 Phase II GAUSS study, in patients with high cholesterol who cannot tolerate statins. Reductions of up to 51 percent were observed in low density lipoprotein cholesterol (LDL-C), or "bad" cholesterol, with AMG 145 and 63 percent with the combination of AMG 145 and ezetimibe, compared to 15 percent with ezetimibe alone. ...

Epanova (Omthera Pharma) successes in EVOLVE and ESPIRIT trials for Dyslipidaemia

Omthera Pharmaceuticals, announced that Phase III results from its EVOLVE (EpanoVa fOr Lowering Very High triglyceridEs) and ESPRIT (Epanova combined with a Statin in Patients with HypertrRglycerIdemia to Reduce Non-HDL CholesTerol) clinical trials for Epanova met all primary and secondary endpoints. Highlights: • Epanova significantly lowers triglycerides (TG) and reduces Non-HDL-C, widely believed to be the most accurate predictor ...

FDA grants accelerated approval for Synribo ( Cephalon/Teva) for CML

The FDA on 26 October 2012 granted accelerated approval for Synribo (omacetaxine mepesuccinate)from Cephalon/Teva to treat adults with Chronic Myelogenous Leukemia (CML), a blood and bone marrow disease.

The drug’s effectiveness in chronic phase CML was demonstrated by a reduction in the percentage of cells expressing the Philadelphia chromosome genetic mutation found in most CML patients. Fourteen out of ...

Successsful Phase III study of Actimid in Multiple Myeloma

A phase III, multi-center, randomized, open-label study (MM-003) of Actimid (pomalidomide) plus low-dose dexamethasone compared to high-dose dexamethasone in patients with relapsed and/or refractory Multiple Myeloma was reviewed by a data safety monitoring board (DSMB). The DSMB determined MM-003 met the primary endpoint of improvement in progression-free survival (PFS) at the PFS final analysis. Additionally, at the OS interim ...

FDA Advisory Committee recommends Kynamro for treatment of Homozygous Familial Hypercholesterolemia

Genzyme, a Sanofi company and Isis Pharmaceuticals have announced that the Endocrinologic and Metabolic Drugs Advisory Committee of the FDA voted 9 to 6 that Genzyme had provided sufficient efficacy and safety data to support the marketing of Kynamro (mipomersen sodium) for the treatment of patients with Homozygous Familial Hypercholesterolemia (HoFH). Many people with HoFH have aggressive cardiovascular disease beginning ...

FDA Advisory Committee recommends lomitapide (Aegerion Pharma) for HoFH

In a 13-2 vote, the FDA Endocrinologic and Metabolic Drugs Advisory considered that Aegerion Pharma provided sufficient efficacy and safety data to support marketing of lomitapide for Homozygous Familial Hypercholesterolemia (HoFH), despite its association with liver toxicity. The application was based on a single 18-month open-label, single-arm pivotal Phase III trial, which only enrolled 29 patients with HoFH, with supportive ...

Turoctocog alfa (Novo Nordisk) filed for Haemophilia in US and EU

Novo Nordisk announced the submission of the regulatory application for turoctocog alfa (NN7008) to the European Medicines Agency (EMA) and the FDA. Turoctocog alfa is a third-generation recombinant coagulation factor VIII intended for prevention and treatment of bleeding in people with haemophilia A.

The decision to apply for marketing authorisation for turoctocog alfa is based upon the results ...

EU approves subcutaneous Velcade (Janssen) for treatment of Multiple Myeloma

The EC has approved the subcutaneous version of Velcade (bortezomib) from J&J Janssen Cilag for Multiple Myeloma. Subcutaneous bortezomib has fewer side effects and offers greater convenience for patients, with similar efficacy compared to intravenous bortezomib....

Novo Nordisk cancels further development of vatreptacog alfa as a treatment for Diabetes

Novo Nordisk has announced the decision to discontinue the development of vatreptacog alfa, a fast-acting recombinant factor VIIa analogue for haemophilia patients with inhibitors. The decision follows analysis of the data from the phase IIIa trial adeptTM 2. A few patients in the trial had developed anti-drug antibodies to vatreptacog alfa, one patient with a potentially neutralising effect....

EU approval for Dacogen (Janssen Cilag) for treatment of Acute Myeloid Leukemia

Astex Pharmaceuticals has announced that Janssen-Cilag International NV was notified that the European Commission has approved the marketing authorization for Dacogen (decitabine) for the treatment of adult patients (age 65 years and above) with newly diagnosed de novo or secondary Acute Myeloid Leukemia (AML), according to the World Health Organization (WHO) classification, who are not candidates for standard induction ...

Belinostat (TopoTarget/Spectrum) success in BELIEF study for PTCL

The pivotal BELIEF trial (PXD101-CLN-19) evaluating the efficacy and safety of belinostat from Topo Target A/S for the treatment of patients with relapsed/refractory Peripheral T-Cell Lymphoma (PTCL) met its primary endpoint. A Special Protocol Assessment agreement with the FDA requires the BELIEF trial to reach an objective response rate (ORR) of at least 20% and this objective was met. Data ...

Successful Phase III trial for rFIXFc (Biogen/SOBI) for Haemophilia

Top-line results from B-LONG, a Phase III 123-patient study of the long-lasting recombinant Factor IX Fc fusion protein (rFIXFc), from Biogen IDEC and SOBI, showed that the treatment was effective in the control and prevention of bleeding, routine prophylaxis and perioperative management. Indeed, one injection of rFIXFc controlled 90.4% of bleeding episodes and the treatment was well-tolerated.

Biogen and SOBI ...

Baxter acquires EU licence to rigosertib from Oncanova

Baxter International Inc. and Onconova Therapeutics Inc.have announced that they have entered into a European licensing agreement for rigosertib, a novel targeted anti-cancer compound from Onconova currently in a Phase III study for the treatment of a group of rare hematologic malignancies called Myelodysplastic Syndromes and in a Phase II/III study for Pancreatic Cancer. Under the terms of the agreement, ...

Baxter files BAX 326 at FDA for management of Haemophilia B

Baxter has announced that the company has submitted a biologics license application (BLA) to the FDA for approval of BAX 326, a recombinant factor IX (rFIX) protein being investigated for the treatment and prophylaxis of bleeding episodes for patients over 12 years of age with Haemophilia B. Haemophilia B, also known as Christmas disease, is the second most common type ...

FDA approves Bosulif (Pfier) for treatment of PH+CML

Pfizer Inc. has announced that the FDA has approved Bosulif (bosutinib),an Abl and Src kinase inhibitor, for the treatment of adult patients with chronic, accelerated, or blast phase Philadelphia chromosome-positive (Ph+) Chronic Myelogenous Leukemia (CML) with resistance, or intolerance to prior therapy. Patients in the registrational trial included patients who were previously treated with imatinib [Gleevec] or imatinib plus at ...

Ariad Pharma files ponatinib at EMA for CML and Ph+ALL

Ariad Pharmaceuticals has submitted its marketing authorisation application to the European Medicines Agency for ponatinib for resistant or intolerant Chronic Myeloid Leukaemia and Philadelphia-chromosome positive Acute Lymphoblastic Leukaemia.The drug was filed in the US for these indications in July 2012....

Xarelto(Bayer/Janssen) sub group analysis of TIMI trial shows reduction in mortality for ACS patients

A major subgroup analysis from the ATLAS ACS 2-TIMI 51 study of 7,817 patients with ACS and a recent ST-segment elevation myocardial infarction (STEMI) demonstrated that oral anticoagulant Xarelto (rivaroxaban) 2.5 mg twice daily, from Bayer/Janssen, when added to standard antiplatelet therapy, provides a significant mortality benefit. The analysis showed that adding rivaroxaban to standard antiplatelet therapy significantly reduced the ...

EU Commission approves Jakavi (Novartis) for Myelofibrosis

Novartis received approval from the European Commission for Jakavi (INC424, ruxolitinib), a JAK 1 and JAK 2 inhibitor for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (also known as Chronic Idiopathic Myelofibrosis), post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. The European Commission's decision was based on positive findings from the COMFORT (COntrolled MyeloFibrosis Study ...

Review finds insufficient evidence for effectiveness of Cytotec(Pfizer) for preventing PPH

A review finds that there is insufficient evidence for the effectiveness of Cytotec (misoprostol), from Pfizer, a drug increasingly used in low- and middle-income countries for preventing postpartum haemorrhage (PPH). It is given to women during Labour to prevent uncontrolled bleeding, and it is included on the WHO's Essential Medicines List for this use.

However, researchers led by Professor Allyson Pollock ...

Campath/MabCampath (Sanofi/Genzyme) withdraws marketing in EU and US for oncology indications.

Sanofi/Genzyme have withdrawn MabCampath (alemtuzumab) from the European market and established a free distribution programme for B-cell CLL users. In the United States the FDA licence will be maintained but a free Campath Distribution Program will offer the drug freely to patients. The intention is to avoid confusion between Campath/MabCampath and the newly developed version of alemtuzumab for which Sanofi/Genzyme ...

FDA approves Marqibo (Talon Therapeutics) for Ph-ALL

The FDA has approved the orphan drug Marqibo (vincristine sulfate liposomes injection)from Talon Therapeutics, as a treatment for patients with Philadelphia chromosome negative (Ph-) Acute Lymphoblastic Leukemia (ALL). Marqibo is approved for patients whose leukemia has returned (relapsed) two or more times, or whose leukemia has progressed following two or more regimens of anti-leukemia therapy.

Marqibo contains vincristine, a ...

ODYSSEY Phase III trials enroll for SAR 236553 treatment of LDL Cholesterol

Sanofi and Regeneron Pharmaceuticals, Inc. announced that several trials within ODYSSEY, the Phase III clinical program of SAR236553/REGN727, have initiated patient enrollment. SAR236553/REGN727 is a potential first-in-class, subcutaneously administered, fully-human antibody that lowers low-density lipoprotein (LDL) cholesterol by targeting PCSK9 (proprotein convertase subtilisin/kexin type 9), an enzyme which binds LDL receptors, leading to their accelerated degradation and increased LDL-cholesterol (LDL-C) ...

CHMP recommends Adcetris ( Takeda) for CD 30+ Hodgkin Lymphoma

On 19 July 2012, the CHMP adopted a positive opinion, recommending the granting of a conditional marketing authorisation for the medicinal product Adcetris (brentuximab vedotin) from Takeda/Millennium/Seattle Genetics, 50 mg, powder for concentrate for solution for infusion, intended for the treatment of of adult patients with relapsed or refractory CD30+ Hodgkin Lymphoma (HL): (1) following autologous stem cell transplant (ASCT) ...

CHMP recommends Dacogen (Janssen/Eisai) for Acute Myeloid Leukemia

On 19 July 2012, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Dacogen, 50 mg, powder for concentrate for solution for infusion, from Eisai/Janssen and intended for the treatment of Acute Myeloid Leukaemia. Dacogen was designated as an orphan medicinal product on 8 June ...

CHMP refuses recommendation of Istodax( Celgene) for T-cell Lymphoma

On 19 July 2012, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a conditional marketing authorisation for the medicinal product Xalkori (crizotinib), 200 mg, 250 mg, hard capsule from Pfizer and intended for the treatment of adults with previously treated anaplastic lymphoma kinase (ALK)-positive advanced Non-Small Cell Lung Cancer (NSCLC) . ...

FDA approves Kyprolis (Onyx Pharma) for Multiple Myeloma

The FDA has approved Kyprolis (carfilzomib) from Onyx Pharma to treat patients with Multiple Myeloma who have received at least two prior therapies, including treatment with Velcade (bortezomib) and an immunomodulatory therapy. A form of blood cancer that arises from plasma cells, Multiple Myeloma usually grows in bone marrow, the soft, spongy tissue found inside most bones. The bone marrow ...

Neupro (UCB/Schwarz) re inrroduced in US for RLS and Parkinsons Disease

Neupro(rotigotine transdermal patch) from UCB/Schwarz is now available again in the US after getting release from the FDA in April to treat the signs and symptoms of early and advanced stage idiopathic Parkinsons Disease and moderate-to-severe primary Restless Legs Syndrome. Neupro is a once-daily patch that provides continuous delivery of the dopamine agonist rotigotine for 24 hours. The treatment was ...

Positive results from Phase III trial of PA21 (Vifor Pharma) for controlling Hyperphosphataemia

The pivotal Phase III clinical study shows that PA21 (iron oxyhydroxide), from Vifor Pharma, successfully controls Hyperphosphataemia in patients with chronic Kidney Disease on dialysis. Results of the 6-month, study with more than 1,000 patients conducted in the USA, Europe as well as in Russia, Ukraine and South Africa established the superiority of maintenance doses of PA21 versus a PA21 ...

FDA halts trials of IB 1001 (Inspiration Pharmaceuticals) for Haemophilia B

The FDA has placed a hold on clinical trials evaluating the safety and efficacy of IB 1001, an intravenous recombinant factor IX (rFIX), from Inspiration Pharmaceuticals, being investigated for the treatment and prevention of bleeding episodes in people with Haemophilia B. Trials have shown a higher proportion of individuals treated with IB 1001 have developed antibodies to proteins from the ...

Janssen Cilag withdraws application at EMA for Velcade in Follicular Lymphoma

Janssen Cilag has withdrawn its application for EU approval for the use of Velcade (bortezomib) in combination with rituximab as a treatment for patients with relapsed Follicular non-Hodgkin Lymphoma. The European Medicines Agency said it had been formally notified by the company that it was withdrawing the application because the Committee for Medicinal Products for Human Use indicated that the ...

Exembol (Mitsubishi Tanabe) launched in UK for HIT

Mitsubishi Tanabe Pharma's (MTP) has launched the direct thrombin inhibitor Exembol (argatroban) in the UK for the treatment of adult patients with Heparin-Induced Thrombocytopenia type II (HIT) who require parenteral antithrombotic therapy. Exembol is already launched in Netherlands, Germany/Austria/Scandinava and is marketed in the US by GSK....

Ixazomib (Takeda/Millennium) enters Phase III trials for Multiple Myeloma

Takeda and Millennium Oncology have begun a Phase III development programme for ixazomib citrate (MLN 9708), the first oral proteasome inhibitor to enter clinical trials for Multiple Myeloma and to be a possible successor to the Velcade. The trial is TOURMALINE-MM1 which will assess MLN 9708 in patients with relapsed and/or refractory Multiple Myeloma at sites in North and South ...

FDA approves Verigene GP Blood Culture Nucleic Acid Test (Nanosphere) for identifying Bacterial Infections

The FDA has allowed marketing of the Verigene GP Blood Culture Nucleic Acid Test (BC-GP), from Nanosphere. This is the first nucleic acid test that can identify 12 different bacterial types known to cause bloodstream infections. The test allows for simultaneous identification of the bacteria and three associated resistance genes in just a few hours after the first sign of ...

Positive results for Exjade (Novartis) in patients with Iron Overload

A new clinical study of Exjade (deferasirox), from Novartis, as a treatment for patients with Myelodysplastic Syndrome (MDS) a blood-related malignancy that involves the ineffective production of blood cells, shows beneficial iron reduction. The majority of patients with MDS require red blood cell transfusions for their anemia, but this leaves them at risk for iron overload. The results of this ...

CHMP recommends subcutaneous Velcade (Janssen) for Multiple Myeloma

The CHMP has granted a positive opinion recommending approval of subcutaneous administration of Velcade (bortezomib), from J&J Janssen Cilag, for patients with Multiple Myeloma. Subcutaneous bortezomib has fewer side effects and offers greater convenience for patients, with similar efficacy compared to intravenous bortezomib...

FDA Advisory Committee recommends Kyprolis(Onyx) for Multiple Myeloma

Onyx Pharmaceuticals announced that the FDA Oncologic Drugs Advisory Committee determined by a vote of 11-0 (with 1 abstention) that, in patients with relapsed and refractory Multiple Myeloma who have received at least two prior lines of therapy that included a proteasome inhibitor and an immunomodulatory agent, the benefit-risk assessment is favorable for the use of Kyprolis (proposed brand name ...

CHMP requires more data for Revlimid (Celgene) application for maintenance therapy in Multiple Myeloma

Celgene has decided to withdraw the new indication submission to the Committee for Medicinal Products for Human Use (CHMP) for Revlimid (lenalidomide), which was intended for the maintenance treatment of newly diagnosed Multiple Myeloma patients who have not progressed following initial treatment with melphalan, prednisone and Revlimid (lenalidomide), or maintenance therapy following autologous stem cell transplantation. In response to the ...

Three-year follow-up data on Sprycel (Otsuka/BMS) shows fast, deep response in CML patients

Results from the 3-year follow-up of the DASISION trial shows that first-line treatment with Sprycel (dasatinib), from BMS and Otsuka, results in faster and deeper response rates compared with Gleevec in patients with newly diagnosed Philadelphia+ Chronic Myeloid Leukaemia. An exploratory analysis of the study suggests that patients with a deeper molecular response at three months (defined as having a ...

New results for Livazo(Kyowa Hakko Kirin) in Dyslipidaemia

New results show Lival/Livazo (pitavastatin)from Kyowa Hakko Kirin is as effective as other commonly used statins at lowering low density lipoprotein cholesterol (LDL-C), with excellent tolerability. It is also effective at elevating high density lipoprotein (HDL-C) levels, reducing triglyceride levels, and appears to have less effect on glycaemic control, than some other statins. Comparisons to simvastatin in patients with dyslipidemia ...

Six-year follow up on Phase III study of Sprycel (BMS / Otsuka) shows positive results for CML patients

Results from a Phase III study of Sprycel (dasatinib), from BMS and Otsuka, in Philadelphia chromosome-positive (Ph+) chronic-phase chronic myeloid leukaemia (CP-CML) adult patients shows progression-free survival of 49.3% and an overall survival of 71% with 6% of patients progressing to accelerated or blast phase on study at six years of follow-up. Study CA180-034 was designed to assess the efficacy ...

FDA reject Taltorvic (Merck Inc) for Sarcomas

The FDA has issued a complete response letter to an application for Sarcoma drug Taltorvic (ridaforolimus), from Merck Inc., saying it cannot approve the application in its present form, and "that additional clinical trial(s) would need to be conducted to further assess safety and efficacy". The rejection follows a vote in March in which the agency's Oncologic Drugs Advisory Committee ...

Treanda (Teva/Mundipharma) success in NHL study

Treanda/Levact(bendamustine) from Teva/Mundipharma/Napp combined with Rituxan/MabThera outperformed the current standard of care in treating Non-Hodgkin's Lymphoma (NHL).In the trial, which included more than 500 patients, Treanda slowed NHL growth for 55 months, compared with just 35 months in those patients taking the standard four-drug treatment. After 32 months, 40 percent of Treanda patients experienced full remission, while 31 percent went ...

Treanda(Teva/Mundipharma)success in NHL study

Treanda/Levact(bendamustine) from Teva/Mundipharma/Napp combined with Rituxan/MabThera outperformed the current standard of care in treating Non-Hodgkin's Lymphoma (NHL).In the trial, which included more than 500 patients, Treanda slowed NHL growth for 55 months, compared with just 35 months in those patients taking the standard four-drug treatment. After 32 months, 40 percent of Treanda patients experienced full remission, while 31 percent went ...

PREVAIL trial shows positive effects of Livalo(Kowa Hakko Kirin) over pravastatin in LDL-C

Kowa Pharmaceuticals America, Inc. and Eli Lilly and Company have announced results of the PREVAIL U.S. study which evaluated the efficacy of Livalo (pitavastatin) 4 mg compared with pravastatin 40 mg in reducing low-density lipoprotein cholesterol (LDL-C), the primary endpoint, as well as effects on other lipid parameters and lipoprotein particles in adult patients with primary hyperlipidemia or mixed Dyslipidemia. ...

EMA finds MabThera (Genentech/Roche) safe to use.

The EMA has cleared MabThera (rituximab), from Genentech/Roche, as safe after an assessment found that contamination at the manufacturer's bioreactors poses little threat. This follows a contamination scare at Roche's facility in Vacaville, California, US. MabThera is approved to treat Non-Hodgkin's Lymphoma and Chronic Lymphocytic Leukaemia, as well as Rheumatoid Arthritis....

Positive data from PIX301 trial of Pixuvri (Cell Therapeutics) for Non Hodgkin Lymphoma

New results have been released from the Phase III trial of Pixuvri (pixantrone dimaleate), from Cell Therapeutics, for patients with relapsed or refractory, aggressive Non Hodgkin Lymphoma who received two or more prior therapies and who were sensitive to treatment with anthracyclines. The PIX301 trial enrolled 140 patients who were randomized to receive either Pixuvri or another single-agent drug. Pixuvri ...

Tasgina updates of ENEST programme in Ph+CML presented at ASCO meeting

Two Phase III studies from the ENEST clinical research program, demonstrate that twice as many adult patients with Philadelphia chromosome-positive Chronic Myeloid Leukemia (Ph+ CML) in chronic phase treated with Tasigna (nilotinib) from Novartis achieved deeper levels of response compared to those treated with imatinib. These findings were first presented at the 2011 American Society of Hematology Annual Meeting and ...

FDA accepts BLA for human 4-factor prothrombin complex concentrate (CSL Behring) for acute Bleeding

The FDA has accepted a Biologics License Application (BLA) for a human 4-factor prothrombin complex concentrate (PCC), from CSL Behring, for the urgent reversal of vitamin K-antagonist therapy (i.e., warfarin) in patients with acute major Bleeding. The submission is based on results from three prospective Phase III clinical trials that evaluated the safety and efficacy of PCC in patients who ...

SAR 236553 success in Phase II study for heFH

Sanofi and Regeneron have announced results from study 1003 of SAR 236553/REGN 727, a subcutaneously administered, fully-human antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9) intended to treat Heterozygous Familial Hypercholesterolemia (heFH). The trial randomized 77 patients with heFH whose LDL-cholesterol (LDL-C) levels remained uncontrolled on statin therapy with or without ezetimibe. Across the four different dosing regimens tested, patients ...

CHMP recommends rFXIII (Novo Nordisk) for FXIII A-subunit Deficiency

Novo Nordisk has announced that the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion on the recombinant factor XIII product (rFXIII). The Committee recommended marketing authorisation for rFXIII for once-monthly replacement therapy in patients from the age of six years with congenital factor XIII A-subunit deficiency. Congenital factor XIII deficiency is a rare bleeding disorder with ...

Positive data from VOYAGER trial of Crestor (AstraZeneca) for Dyslipidemia patients

Data presented from the VOYAGER study demonstrate the benefits of aggressive statin therapy with Crestor (rosuvastatin), from AstraZeneca, in comparison to atorvastatin and simvastatin in high risk Dyslipidemia patients. VOYAGER is an individual patient data meta-analysis of 32 258 patients from 37 randomised studies, comparing the effects of statin therapy with Crestor (rosuvastatin) to that of atorvastatin and simvastatin. The ...

Lancet study shows raising HDL may not affect Heart Disease risk

A new study published in The Lancet has found that raising HDL levels may not make a difference to heart disease risk. People with genes that result in higher HDL levels have no less heart disease (myocardial infarction) than those who inherit genes that give slightly lower levels. If HDL were protective those with genes causing higher levels should have ...

Revlimid (Celgene) prolongs disease control for Multiple Myeloma patients

Multiple myeloma patients are better equipped to halt progression of this blood cancer if treated with Revlimid (lenalidomide), from Celgene, following a stem cell transplant. A Phase III study, co-authored by Richard Maziarz, M.D., of the OHSU Knight Cancer Institute, found a 63 percent reduction in the risk of progressive myeloma or death for the stem cell transplant patients that ...

IMDx GBS (IntelligentMDx) test system for Group B Streptococcus receives CE mark

The IMDx GBS for Abbott m2000, from IntelligentMDx, an automated, high-throughput, qualitative in vitro diagnostic test designed for the rapid detection of Group B Streptococcus (GBS) for use in screening pregnant women and those in labor who may be infected with the pathogen, has received CE mark approval. The system utilizes real time PCR, operates on the Abbott m2000 system ...

Pixuvri (Cell Therapeutics) approved in EU to treat adults with Non-Hodgkin B-Cell Lymphomas

The European Commission has given conditional marketing authorization for Pixuvri (pixantrone), from Cell Therapeutics, as monotherapy for the treatment of adult patients with multiply relapsed or refractory aggressive Non-Hodgkin B-cell Lymphomas. Approval is based on the PIX 301 Phase III trial which demonstrated that a greater proportion of patients achieved a complete response or unconfirmed complete response to Pixuvri than ...

Roche halts development of RG 1658 for Dyslipidaemia

Roche has halted development of RG 1658 (dalcetrapib) due to its lack of efficacy in treating Dyslipidaemia. This decision is based on results from OUTCOMES, a trial that has been halted after RG 1658 failed to show any benefit for patients already experienced symptoms of coronary artery obstruction taking the drug plus existing standard of care, though there were no ...

F 627 (Generon) commences Phase II study for Neutropenia

A Phase II clinical trial for F 627,recombinant G-CSF from Generon(Shanghai)Corporation, has been started after FDA regulatory clearance. The study is a randomized, multicenter, open-label, active-controlled trial of F 627 in women with Breast Cancer receiving myelotoxic chemotherapy. The clinical trial will enroll approximately 200 patients and will be conducted at approximately 35 clinical centers in North America and Europe. ...

US Court rules against Mylan that Zetia (Merck Inc.) patent is valid

A US Federal judge has ruled in two jointly related patent infringement suits against Mylan that a patent on hypolipaemic Zetia (ezetimibe) and related product Vytorin (ezetimibe plus simvastatin) from Merck Inc. is valid and has issued an injunction blocking the approval of generic versions by Mylan until the patent at issue expires 25 April 2017. Merck Inc.has agreed with ...

EU approves Signifor (Novartis) for Cushing's Disease

Novartis announced that the European Commission has approved Signifor (pasireotide) for the treatment of adult patients with Cushing's Disease for whom surgery is not an option or for whom surgery has failed. Signifor is the first medicine to be approved in the European Union (EU) targeting Cushing's Disease. The approval is based on data from the largest randomized Phase III ...

CHMP recommends Rienso (Takeda) for Iron Deficiency Anaemia

)The CHMP has recommended marketing authorisation for Rienso, 30 /ml, solution for injection from Takeda, being the intravenous treatment of Iron Deficiency Anaemia in adult atients with Chronic Kidney Disease. The active substance of Rienso is ferumoxytol, a colloidal iron-carbohydrate complex. Upon release from the complex, the iron either enters the intracellular storage iron pool (e.g., ferritin) or is transferred ...

Cell Therapeutics acquires world rights to pancritinib for Myelofibrosis

Cell Therapeutics has acquired a worldwide licence from Singapore-based S*Bio to develop and manufacture pancritinib, an oral once daily, tyrosine kinase inhibitor with dual activity against JAK 2 (Janus Associated Kinase 2) and FMS-like tyrosine kinase 3 (FLT 3). Pacritinib is in Phase II clinical trials for Myelofibrosis with a Phase III trial planned. The drug has Orphan designation in ...

CHMP recommends Jakavi (Incyte/Novartis)for Myelofibrosis

The CHMP of the European Medicines Agency adopted a positive opinion for Jakavi (INC424, ruxolitinib) from Incyte/Novartis for the treatment of disease-related splenomegaly or symptoms in adult patients with primary Myelofibrosis (also known as chronic idiopathic myelofibrosis), post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Globally, there are few available treatments for myelofibrosis, an uncommon, life-threatening blood cancer characterized by multiple ...

IB 1001 (Inspiration Bio) filed at FDA for Haemophilia B

Inspiration Biopharmaceuticals has submitted a biologics license application (BLA) to the FDA for the approval of IB1001, an intravenous recombinant factor IX for the treatment and prevention of bleeding in Haemophilia B. Ipsen has European rights to the product....

IB 1001 (Inspiration BioPharm) is filed at FDA for Haemophilia B

Inspiration Biopharmaceuticals has submitted a biologics license application (BLA) to the FDA for the approval of IB1001, an intravenous recombinant factor IX for the treatment and prevention of bleeding in Haemophilia B. The product has been filed at EMA by Ipsen and launches are expected in Europe in late 2012 or early 2013 and in the USA in early 2013....

Minophagen extends marketing rights to bexarotene for T-Cell Lymphoma

Minophagen Pharma has acquired further rights to market the synthetic retinoid bexarotene from Eisai. Minophagen has added to its rights to market in Japan the territories of Asia, Oceana, the Middle East and Eastern Europe.The drug is marketed in 20 countries for refractory/recurrent T-Cell Lymphoma and in the USA by Eisai as Targretin....

ETC 1002 (Esperion Therapeutics) positive in Phase II trial for Dyslipidaemia

Esperion Therapeutics, has announced positive results from a Phase II clinical trial for ETC-1002. This novel small molecule activator of AMP Kinase has demonstrated preclinical and clinical activity as a metabolic regulator of imbalances in lipid and carbohydrate metabolism. Results were presented in an oral presentation at the 2012 American College of Cardiology meeting in Chicago....

Kynamro (Genzyme/Sanofi)is filed at FDA for Homozygous Familial Hypercholesterolaemia

Kynamro(mipomersen) from Genzyme/Sanofi and Isis Pharma has been filed at the FDA as a New Drug Application for the rare genetic condition of Homozygous Familial Hypercholesterolaemia. The first-in-class apo-B synthesis inhibitor was filed in Europe in July last year....

FDA approves Omontys(Affymax/Takeda)for anaemia for patients on Dialysis

The FDA has approved Omontys (peginesatide) from Affymax Inc.,/Takeda to treat Anaemia, a condition in which the body does not have enough healthy red blood cells, in adult dialysis patients who have chronic kidney disease (CKD).Omontys is a new erythropoiesis-stimulating agent (ESA) that aids in the formation of red blood cells. It works by stimulating the bone marrow to produce ...

FDA Advisory Committee recommends Marqibo for Ph-ALL

Talon Therapeutics has announced the FDA Oncologic Drugs Advisory Committee voted 7 yes, 4 no, and 2 abstain that evidence from clinical studies supports a favorable benefit/risk assessment for use of Marqibo(vincristine sulfate liposomes injection) for the indication for the treatment of adult Philadelphia chromosome-negative (Ph-) Acute Lymphoblastic Leukemia (ALL) in second or greater relapse or that has progressed following ...

AMG 145 success in Phase 1b trial for High Cholesterol

Amgen has announced positive results from a Phase 1b clinical study of AMG 145, an investigational PCSK9 inhibitor, in patients with high cholesterol who were taking statins. The study demonstrated that multiple doses of AMG 145 significantly reduced serum low density lipoprotein cholesterol (LDL-C), also known as "bad" cholesterol, by up to 81 percent versus placebo (maximum reduction) in subjects ...

Phase II success of SAR236553/REGN727 for Cholesterol Lowering

Regeneron/Sanofi announced data from two Phase II trials with SAR236553/REGN727, an investigational, high-affinity, subcutaneously administered, fully-human antibody targeting PCSK9 (proprotein convertase subtilisin/kexin type 9), were presented at the American College of Cardiology’s (ACC) 61st Annual Scientific Meeting in Chicago. The data showed that treatment with SAR236553/REGN727 over 8 to 12 weeks significantly reduced mean low-density lipoprotein-cholesterol (LDL-C, or “bad” cholesterol) ...

FDA advisors vote against Taltorvic (Merck/Ariad) for Sarcoma patients

The FDA's ODAC voted 13-1 against the use of Taltorvic (ridaforolimus), from Merck / Ariad, as maintenance therapy for patients with metastatic soft-tissue Sarcoma or Bone Sarcoma whose disease has not progressed after at least four cycles of chemotherapy. This was based on data showing patients on Taltorvic had a median progression-free survival of 17.7 weeks compared with 14.6 weeks ...

NICE recommends Tasigna (Novartis) for CML

In final draft guidance, NICE has recommended Tasigna (nilotinib), from Novartis, for CML. The decision is not based on direct data. The independent appraisal committee concluded from indirect comparisons that the drug is effective in treating CML. The panel found that the most plausible Incremental Cost-effectiveness Ratio (ICER) for Tasigna compared with standard-dose Glivec was considered to be £11,000 per ...

NICE does not recommend Sprycel (BMS) for CML

In final draft guidance, NICE has not recommended Sprycel (dasatinib), from BMS, as first-line treatment of Chronic Myeloid Leukaemia. The panel found that the most plausible Incremental Cost-effectiveness Ratio (ICER) for Sprycel compared with standard-dose Glivec exceeded £200,000 per QALY gained....

Purocin protein technology (AvidBiotics) offers rapid response to E.coli infection

A new study shows that AvidBiotics' antibacterial Purocin protein technology can be used to create a highly targeted bactericidal protein against the life-threatening foodborne E. coli O104 strain by making use of rapidly acquired, published, draft genomic sequence data. This strategy offers a rapid-response platform with the potential to create targeted agents for use against emerging bacterial pathogens within days-to-weeks ...

APG101 (Apogenix) meets primary endpoint in Phase II trial for Glioblastoma Multiforme

The Phase II clinical efficacy trial with APG101, from Apogenix, has met its primary endpoint in the 2nd line treatment of a common form of Brain Cancer, Glioblastoma Multiforme (GBM), following a six month follow up of the last patient treated. The primary endpoint of the trial was the six-month-rate of progression-free survival (PFS6) and secondary endpoints include overall survival ...

EMA accepts MAA for Hematide (Takeda) for Anaemic adults on dialysis

The EMA has accepted Hematide (peginesatide), from Takeda, for assessment for a Marketing Authorisation Application (MAA) for the treatment of symptomatic Anaemia associated with Chronic Kidney Disease (CKD) in adult patients on dialysis. The submission was supported mainly by data from two Phase III studies (EMERALD 1 and 2) that evaluated the drug's efficacy and safety, dosed once every four ...

Phase I/III study of rVIII-SingleChain (CSL Behring) for Haemophilia A begins

Screening has begun in the AFFINITY clinical trial program for rVIII-SingleChain (rFVIII or recombinant coagulation single-chain factor VIII), from CSL Behring. The rVIII-SingleChain is a novel molecule being studied for the treatment of Haemophilia A. This Phase I/III study examines the crossover safety, efficacy and pharmacokinetics of rVIII-SingleChain compared with recombinant human antihemophilic factor VIII (octocog alpha). In Part 1 ...

Enrollment complete for Phase III Carfilzomib (Onyx Pharmaceuticals) trial for Multiple Myeloma

The Phase III ASPIRE trial evaluating carfilzomib, from Onyx Pharmaceuticals, in combination with Revlimid (lenalidomide) and low dose dexamethasone in patients with relapsed Multiple Myeloma has reached its target enrollment figures of 780 patients and is being conducted at approximately 200 sites in North America, Europe, and Israel. The FDA is currently reviewing a New Drug Application for potential accelerated ...

CHMP recommends Pixuvri (CTI Life Sciences) for Non Hodgkin's B-cell Lymphoma

The CHMP has given a conditional recommendation for the approval of Pixuvri (pixantrone dimaleate) from CTI Life Sciences for relapsed or refractory aggressive non-Hodgkin’s B-cell Lymphoma. The drug application at the FDA was withdrawn as additional information was requested. The drug is now scheduled to be considered at the FDA Advisory Comitteee meeting in April 2012....

Nordic rights to ReFacto and BeneFIX are returned to Pfizer

Pfizer has taken back the Nordic territory rights to reclaim to the blood clotting factors ReFacto and BeneFIX from Swedish Orphan Biovitrum which held co-promotion rights there.The drugs will continue to be manufactured in Sweden....

Early results for SM101 (Suppremol) are encouraging for ITP

Interim results from a Phase 1b/IIa study of SM 101 from Munich-based SuppreMol GmbH for the treatment of primary Immune Thrombocytopenia were encouraging. SM 101 is a recombinant, soluble, non glycosylated version of the Fc receptor IIb. The protein binds to autoantibody/autoantigen complexes and blocks the activation of Fc receptors on the surface of immune cells. As a result, the ...

Epanova (Omthera) has better bioavailability than Lovaza

Omthera Pharmaceuticals has reported positive data showing that its prescription-grade omega-3 fatty acid candidate, Epanova, has better bioavailability than Lovaza (omega-3 ethyl esters) – the only FDA-approved omega-3 acid drug on the US market from GSK. The latest reported data from a 52-patient pharmacokinetic study showed that after 14 days of dosing and with a low fat diet, plasma EPA ...

FDA approves wider indication for fibrin sealant Tisseel (Baxter)

The FDA has approved Tisseel [Fibrin Sealant] from Baxter to include general hemostasis in surgery when control of bleeding by standard surgical techniques is ineffective or impractical.TISSEEL mimics the final stages of the body’s own blood clotting cascade, creating a clot that adheres to the wound surface and helps achieve hemostasis....

Pfizer files bosutinib at FDA for PH+CML

The FDA has accepted its New Drug Application (NDA) for standard review of bosutinib, a once a day oral dual Src and Abl kinase inhibitor from Pfizer, as a treatment option for adult patients with previously treated Philadelphia chromosome positive (Ph+) chronic myeloid leukemia (CML). This submission was based on efficacy and safety data from Study 200, a single-arm study ...

Spectrum Pharma acquires world rights to Zevalin for NHL

Spectrum Pharmaceuticals will acquire from Bayer Healthcare the licensing rights to market Zevalin (ibritumomab tiuxetan) injection for intravenous use for the non-Hodgkin's lymphoma treatment outside the USA. Spectrum already has the rights to market the drug in the USA and will now hold the worldwide rights to Zevalin. The product is currently approved in more than 40 countries outside the ...

CHMP rejects Folotyn (AllosTherapeutics and Mundipharma) for T-Cell Lymphoma

The CHMP adopted a negative opinion, recommending the refusal of marketing authorisation for the medicinal product Folotyn,(pralatrexate injection), from Allos Therapeutics, intended for the treatment of peripheral T-cell Lymphoma. The CHMP was concerned that the main study was designed in a way that did not allow the Committee to assess the benefit of the medicine, particularly since Folotyn was not ...

New FDA drug labels for anaemia associated with Chronic Kidney Disease patients treated with ESAs

The FDA recommends caution when prescribing drugs that treat Anaemia for patients with Chronic Kidney Disease (CKD) since the drug label's recommendations fall short. The recommendations apply to erythropoiesis-stimulating agents (ESAs) such as Epogen and Procrit (epoetin alfa) and Aranesp (darbepoetin alfa). Trials show that ESAs can increase the risk of heart-related problems such as strokes and heart attacks when ...

CHMP recommends Signifor (Novartis) for treatment of Cushing's Disease

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for Signifor (SOM230, pasireotide)from Novartis for the treatment of Cushing's Disease. There are currently no approved medicines in the European Union targeting Cushing's Disease, a debilitating endocrine disorder caused by excess cortisol in the body due to the presence of ...

Pfizer will not proceed with Lyrica for Restless Legs Syndrome

Pfizer will not proceed with regulatory approval for Lyrica (pregabalin), its GABA analogue, for Restless Legs Syndrome despite reporting positive Phase III results for the drug in this patient group....

FDA approves Advate (Baxter) for prophylaxis of Hemophilia A

The FDA has approved Advate [Antihemophilic Factor (Recombinant) Plasma/Albumin Free Method] from Baxter for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with Hemophilia A. Advate is the only antihemophilic factor approved in the United States for prophylactic use in both adults and children. The approval is based on a Phase IV prophylaxis study sponsored ...

Zolinza(Merck Inc.) success in Multiple Myeloma trial

A Phase III study investigating Zolinza (vorinostat) from Merck Inc., in combination with bortezomib, in patients with progressive Multiple Myeloma has met its primary endpoint of improved progression-free survival (PFS) in patients with relapsed and/or refractory forms of the disease.The Phase III results from VANTAGE 088 study were presented at the 53rd Annual Meeting of the American Society of Hematology ...

Zolinza (Merck) shows good response with AML patients

Adding Zolinza (vorinostat), from Merck, a histone deacetylase inhibitor that activates genes to frontline combination therapy for Acute Myeloid Leukemia resulted in an 85 percent remission rate after initial treatment. Seventy five patients in a Phase II study received Zolinza, in addition to the chemotherapy drug cytarabine and idarubicin, an anthracycline antibiotic commonly used as chemotherapy. Zolinza activates suppressed genes ...

Exjade (Novartis) success in Thalessaemia trial

Results from THALASSA, the first pivotal placebo-controlled study examining the benefit of iron chelation with Exjade (deferasirox) from Novartis, in patients with non-transfusion-dependent thalassemia (NTDT), show that Exjade can significantly reduce iron overload. These data were presented today at the 53rd Annual Meeting of the American Society of Hematology in San Diego. THALASSA investigated whether patients with NTDT and iron ...

ENEST studies of Tasgina (Novartis)show superiority to Glivec in PH+CML

ENESTcmr study is the first exploratory randomized trial to investigate the impact of switching adult patients with residual disease after a minimum of two years of treatment with Glivec imatinib)from Novartis to Tasigna (nilotinib)from Novartis to determine if a deeper level of response could be achieved. The study showed that twice as many patients switched to Tasigna 400 mg twice ...

FDA advisers vote in favour of Hematide (Takeda/Affymax) approval for Anaemia

The FDA's Oncologic Drugs Advisory Committee has voted 15 to 1 that Hematide (peginesatide), from Takeda and Affymax, demonstrate a favorable benefit/risk profile for use in the treatment of dialysis patients with Anaemia due to chronic kidney disease (CKD). While the FDA is not bound by the recommendations of its advisory committees, their guidance will be considered by the FDA ...

Pixuvri (CellTherapeutics) is refiled at the FDA for NHL

The FDA has accepted the resubmission of Pixuvri(pixantrone dimaleate) from Cell Therapeutics as a single-agent treatment of patients with relapsed or refractory aggressive non-Hodgkin's lymphoma in patients who failed two or more lines of prior therapy.The FDA has not required an additional trial. The FDA has set 24 April 2012 as the new Prescription Drug User Fee Act action date. ...

Onyx Pharma files carfilzomib at the FDA for Multiple Myeloma

The FDA has accepted a new drug application for carfilzomib from Onyx Pharmaceuticals. Onyx is seeking accelerated approval of carfilzomib, a next-generation proteasome inhibitor, as a treatment for patients with relapsed and refractory Multiple Myeloma. The application will be supported by progression-free survival data from the randomized controlled carfilzomib Phase III ASPIRE trial in relapsed multiple myeloma anticipated in early ...

Generic Lipitor is launched in USA by Ranbaxy

The FDA on 30 November 2011 approved the first generic version of the cholesterol-lowering drug Lipitor (atorvastatin calcium tablets) marketed by Pfizer. Ranbaxy Laboratories Inc., has gained approval to make generic atorvastatin calcium tablets in 10 milligram, 20 mg, 40 mg, and 80 mg strengths.The drug is to launch today and is known as Atorvastatin....

AMR 101 (Amarin Corpn)is filed at the FDA for High Triglycerides

A New Drug Application for AMR101 (ethyl EPA) from Amarin Corporation has been accepted for filing by the FDA. Amarin seeks approval to market and sell AMR 101 in the United States for the indication studied in the MARINE trial—the treatment of patients with very high triglycerides (=500mg/dL). The NDA for AMR 101 is supported by data from both Phase ...

BD MAX Assay (Becton, Dickinson and Company) for MRSA detection is EU approved

The BD MAX MRSA Assay, from BD Diagnostics, a Becton Dickinson company, for rapidly and accurately identifing methicllin-resistant Staphylococcus aureus (MRSA) in patients has received CE mark approval in the EU. The BD MAX is a fully automated, bench-top molecular system designed to perform a broad range of molecular testing. This offers the flexibility and versatility to perform CE/in vitro ...

FDA agrees to new labelling of Zevalin (Spectrum Pharma) to remove bioscan Indium 111

The FDA has agreed that Spectrum Pharma can remove from the labelling of Zevalin (ibritumomab tiuxetan) a requirement for a certain type of bioscan, known as an Indium-111 pretreatment imaging evaluation.Patients undergoing treatment with Zevalin no longer need to be exposed to unnecessary radiation with Indium-111. Zevalin is approved in the USA and EU for Follicular Non Hodgkins Lymphoma. Marketing ...

Erwinase (EUSA) is FDA approved for acute lymphoblastic leukemia

The FDA on 18 November 2011 has approved Erwinase from EUSA Pharma as a component of a multi-agent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity to E. coli-derived asparaginase.Erwinase is currently approved in a number of countries, including Canada, the UK and several European Union member states.Many children with ALL develop ...

Jakafi (Incyte/Novartis) is FDA approved for Myelofibrosis

The FDA on 16 November 2011, approved Jakafi(ruxolitinib),from Incyte/Novartis, the first drug approved to specifically treat patients with the bone marrow disease Myelofibrosis. Jakafi is an inhibitor of JAK 1 and 2 (Janus Associated Kinase) that are involved in regulating blood and immunological functioning. The decision was based on two pivotal trials called COMFORT-1 and COMFORT-2....

Injectafer/Ferinject (Vifor/Luitpold) meets endpoints in two trials for Iron Replacement

Two large, multi-center, randomised, controlled clinical trials of Injectafer (US brand name of Ferinject, ferric carboxymaltose) from Vifor Pharma/Luitpold Pharma,were presented at the American Society of Nephrology’s (ASN) Kidney Week 2011.The first trial, poster FR-PO1394, compared Injectafer to oral iron or i.v.iron and the sevond trial, poster LB-PO3155, compared Injectafer to Venofer. Both trials met their efficacy and safety endpoints. ...

Jakafi (Incyte/Novartis) is FDA approved for Myelofibrosis

The FDA on 16 November 2011, approved Jakafi(ruxolitinib),from Incyte/Novartis, the first drug approved to specifically treat patients with the bone marrow disease Myelofibrosis. Jakafi is an inhibitor of JAK 1 and 2 (Janus Associated Kinase) that are involved in regulating blood and immunological functioning. The decision was based on two pivotal trials called COMFORT-1 and COMFORT-2....

Velcade (Millennium/Janssen Cilag) success in Multiple Myeloma trial

The VISTA trial, compared Velcade, from Millennium and Janssen Cilag, with the established standard of care therapy in 682 patients with previously untreated multiple myeloma. The data showed that patients treated with Velcade, melphalan and prednisone continued to have a statistically significantly longer overall survival than patients treated only with melphalan and prednisone (56.4 versus 43.1 months, p<0.05). The complete ...

Pro-FEIBA study success in prophylaxis in Haemophlia A patients

An investigator-initiated study (Pro-FEIBA) evaluated the prophylactic use of FEIBA [Anti-inhibitor Coagulant Complex] from Baxter, to ascertain if it can achieve a decrease in the frequency of joint and other bleeding events in patients with severe hemophilia A and inhibitors,compared to on-demand therapy. The study reported that patients with severe hemophilia A treated with FEIBA prophylactically during a six-month period ...

FDA approves Ferriprox for Iron Overload in Thalassemia patients

The FDA has approved Ferriprox from ApoPharma a subsidiary of generic manufacturer Apotex Inc, as a treatment for Iron Overload due to blood transfusions in patients with thalassemia when current chelation therapy is inadequate. This treatment can be vital for thalassemia patients of whom approximately one quarter are not able to manage Iron Overload with currently available treatment. Iron Overload ...

BAX 111 starts Phase III for Von Willebrand Disease

Baxter International Inc. announced initiation of a Phase III clinical trial to evaluate the safety and effectiveness of BAX 111, an investigational recombinant von Willebrand factor (rVWF), for the treatment and prevention of bleeding episodes in patients with von Willebrand disease. This condition is the most common type of inherited bleeding disorder, affecting both men and women, the majority of ...

Crestor patent challenged by generic companies

Generic manufacturers have challenged the AstraZeneca "314" patent for Crestor (rosuvastatin) and are appealing a decision that upheld the patent in the lower courts. The appeal is based on inequitable conduct. AstraZeneca and its partner Shionogi are seeking to maintain the patent until 2016. Commentators believe the appeal will be unsuccessful. Crestor was worth $5.7 billion to AstraZeneca in 2010....

MabCampath/Campath plus fludarabine offers improved treatment for CLL

A new study shows that combining the chemotherapy drug fludarabine with the monoclonal antibody MabCampath/Campath(alemtuzumab), from Genzyme, significantly increases progression free survival (PFS) and prolongs the lives of patients who have relapsed with the most common type of leukaemia, chronic lymphocytic leukaemia (CLL), compared with fludarabine alone. The findings suggest that this new drug combination could be a less toxic ...

Higher HDL Cholesterol levels reduces Heart Attack and Stroke risk in Diabetes patients

A new study of more than 30,000 patients with Diabetes shows that increasing levels of high-density lipoproteins (HDL cholesterol) reduced the risk for heart attack and stroke. While there is considerable evidence that reducing the amount of low-density lipoprotein, (LDL cholesterol) can reduce the risk of heart disease, the relationship between HDL cholesterol and heart disease is less clear. The ...

Merck Inc. files ridaforolimus at FDA for Metastatic Sarcomas

Merck Inc. has filed ridaforolimus at the FDA seeking approval of the drug as a treatment for patients with metastatic soft tissue or bone Sarcomas. There have been no new treatments in the US for sarcomas in 20 years. The application is based on results of the SUCCEED trial....

Velcade withdrawn from combination treatment of Follicular Lymphoma

Millennium Pharma, the subsidiary of Takeda specialising in oncology, will withdraw its supplemental new drug application to the FDA seeking a new use of Velcade (bortezomib) in combination with Rituxan (rituximab) from Genentech/Biogen Idec as a treatment for patients with relapsed follicular lymphoma....

Mechlorethamine hydrochloride gel is filed at FDA for Mycosis Fungoides

Yaupon Therapeutics has filed at the FDA an application (NDA) for its gel formulation of mechlorethamine hydrochloride as a topical treatment for early-stage mycosis fungoides, the most common type of cutaneous T-Cell lymphoma (CTCL), a rare form of non-Hodgkin's lymphoma. Mycosis fungoides is the most common type of Cutaneous T-Cell Lymphoma, a rare form of non-Hodgkin's lymphoma. The cause of ...

FDA approves Soliris for Hemolytic Uremic Syndrome

The FDA has approved Soliris (eculizumab) from Alexion Pharma for the treatment of patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare, chronic blood disease that can lead to kidney failure, stroke, heart attack and death. This is the first treatment for aHUS that has been FDA-approved and the first approval for use of Soliris in children. The decision is ...

EMA concludes Revlimid provides positive treatment for Multiple Myeloma

The EMA has confirmed that the benefit-risk balance for Revlimid (lenalidomide), from Celgene, used in combination with dexamethasone to treat adults with Multiple Myeloma remains positive. However it also advises of the risk of new cancers as a result of treatment. This review follows the results of three new studies which show a four-fold increase in new cancers (including solid ...

FDA advisors recommend approval of Ferriprox for Iron Overload

The FDA's Oncologic Drugs Advisory Committee has voted 10 - 2 to recommend that the FDA grant accelerated approval of Ferriprox (deferiprone), from ApoPharma. The drug is an oral iron chelator, for the treatment of patients with transfusional Iron Overload when current chelation therapy is inadequate. This can be vital for Thalassemia patients of whom approximately one quarter are not ...

Affymax and Takeda submit NDA for Hematide

Affymax Inc. and Takeda Pharmaceutical Company Limited announced the submission of a New Drug Application (NDA) to the FDA for the investigational agent peginesatide (formerly known as Hematide) for the treatment of anemia associated with chronic renal failure (CRF) in adult patients on dialysis. The NDA includes data from two Phase III studies (EMERALD 1 and 2) that evaluated the ...

Hematide analyse of EMERALD studies

Affymax, Inc. and Takeda Pharmaceutical Company Limited announced results of additional analyses from two Phase III studies (EMERALD 1 and 2) of the investigational agent, peginesatide (formerly known as Hematide) in chronic renal failure (CRF) patients on dialysis with anemia.The EMERALD studies, showed that once-monthly peginesatide was comparable to epoetin given up to 13 times a month in maintaining hemoglobin ...

Zerenex Phase III trials in Japan underway

Keryx Biopharmaceuticals announced that its Japanese partner, Japan Tobacco and Torii Pharmaceutical Co., a subsidiary of JT, has commenced the Phase III clinical program of Zerenex (ferric citrate) in Japan for the treatment of patients with hyperphosphatemia. Zerenex (ferric citrate), a ferric iron-based phosphate binder, is also in Phase III clinical development in the US for the treatment of hyperphosphatemia ...

Elacyt (Clavis Pharma) gets FDA fast track status for AML

Clavis Pharma announced that the FDA had granted fast track status to Elacyt(elacytarabine) for acute myeloid leukemia (AML). Elacytarabine is currently being evaluated in a randomised Phase III trial (known as the CLAVELA study) comparing it with the investigator's choice of treatment in patients with late-stage AML (i.e. those who have failed two or three previous treatment regimes). The primary ...