News

FDA accepts BLA for Rhucin as a treatment for HAE

The FDA has accepted for review the biologic license application (BLA) for Rhucin (recombinant human C1 esterase inhibitor) as a treatment for acute angioedema attacks in patients with Hereditary Angioedema.

The agency set 16 April 2014 as the Prescription Drug User Fee Act action date.

The BLA for Ruconest in HAE was based on data from a randomized placebo-controlled Phase ...

Phase III data on Gencaro (ARCA biopharma) shows drug effective on genetic subset of Atrial Fibrillation patients

New results of a Phase III trial of Gencaro (bucindolol hydrochloride), from ARCA biopharma, in reducing the incidence of new onset Atrial Fibrillation in patients with advanced heart failure with reduced left ventricular ejection fraction (HFREF) show the drug offers significant benefits to a subset of patients. The study was based on a post-hoc analysis of 2,708 HFREF patients known ...

CHMP recommends approval of Voncento (CSL Behring) for Haemophilia A/Von Willebrand disease

The Committee for Medicinal Products for Human Use (CHMP) has recommended the granting of a marketing authorisation for Voncento (Human coagulation factor VIII), from CSL Behring, to be used for the prevention and treatment of bleeding in patients with Haemophilia A and Von Willebrand disease.

The recommendation is based on the assessment of two main studies, one study investigating the ...

CHMP recommends approval of Lojuxta (Aegerion Pharmaceuticals) for HoFH

The European Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for Lojuxta (lomitapide), from Aegerion Pharmaceuticals, as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without low density lipoprotein (LDL) apheresis in adult patients with Homozygous Familial Hypercholesterolemia (HoFH).

The drug was approved by the FDA in December 2012 under the ...

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, ...

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 ...

Metreleptin sub analysis in paediatric patients with Lipodystrophy

BMS and AstraZeneca announce the results from a 12-month sub-group analysis of a National Institutes of Health (NIH), open-label, long-term research study of metreleptin, an investigational agent for the treatment of metabolic disorders associated with inherited or acquired Lipodystrophy (LD), a rare disease estimated to affect a few thousand people around the world, often with an early age of ...

FDA approves Procysbi (Raptor Pharmaceutical) for Nephropathic Cystinosis

The FDA has approved Procysbi (cysteamine bitartrate) delayed release capsules, from Raptor Pharmaceutical, for the treatment of Nephropathic Cystinosis in adults and children 6 years and older. The approval was based on a New Drug Application (NDA) comprising data from six clinical trials, including a multi-center randomized, active-controlled Phase III trial of 43 patients with Nephropathic Cystinosis and extension data ...

BioMarin files Vimzim at EMA for Mucopolysaccharidosis Type IVA

BioMarin Pharmaceutical Inc. has announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA (MPS IVA), also called Morquio A Syndrome. A Biologics License Application for Vimizim was submitted to ...

Ruconest (Santarus/Pharming) is re-filed with FDA for treatment of Hereditary Angioedema

Santarus, Inc. and Pharming Group NV have announced the submission of a Biologics License Application (BLA) to the FDA to obtain marketing approval for Ruconest (recombinant human C1 esterase inhibitor) 50 U/kg, an investigational drug for the treatment of acute angioedema attacks in patients with Hereditary Angioedema (HAE). The safety and efficacy of Ruconest for the treatment of HAE attacks ...

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. ...

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 ...

FDA approves TOBI Podhaler (Novartis) for treatment of Cystic Fibrosis infections

The FDA has approved TOBI Podhaler (tobramycin inhalation powder) from Novartis on 22 March 2013 for the management of Cystic Fibrosis patients with Pseudomonas aeruginosa, a bacterium that causes lung infections.

TOBI Podhaler was approved in the EU for treatment of pseudomonas lung infection in Cystic Fibrosis patients in July 2011.

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; ...

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 ...

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 approves Argus II Retinal Prosthesis System for Retinitis Pigmentosa

The FDA on 14 February 2013 approved the Argus II Retinal Prosthesis System from Second Sight Medical Products Inc., the first implanted device to treat adult patients with advanced Retinitis Pigmentosa (RP). The device, which includes a small video camera, transmitter mounted on a pair of eyeglasses, video processing unit (VPU) and an implanted retinal prosthesis (artificial retina), replaces ...

Sanofi/Genzyme reports positive results from ENGAGE and ENCORE studies of eliglustat for Gaucher disease type 1

New data is reported from the Phase III ENGAGE and ENCORE studies of eliglustat tartrate from Genzyme/Sanofi, the investigational oral therapy for Gaucher disease type 1. The results from the ENGAGE study were presented at the 9th Annual Lysosomal Disease Network WORLD Symposium in Orlando, Fla. In conjunction with this meeting, Genzyme also released topline data from its second Phase ...

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 approves Ravicti (Hyperion Therapeutics ) for Urea Cycle Disorders

On 31 January the FDA approved Ravicti (glycerol phenylbutyrate) from Hyperion Therapeutics, for the chronic management of some Urea Cycle Disorders in patients ages 2 years and older.

Urea Cycle Disorders, or UCDs, are a collection of inherited metabolic disorders in which affected patients suffer from high levels of systemic ammonia, a potent neurotoxin. Ammonia is produced via ...

Bronchitol (Pharmaxis) is rejected by FDA Advisory Committee as a treatment for Cystic Fibrosis

Pharmaxis has received a negative recommendation from the Pulmonary-Allergy Drugs Advisory Committee (PADAC) advising the FDA on the use of Bronchitol (inhaled dry powder mannitol) for Cystic Fibrosis patients in the United States.

The PADAC voted on three questions relating to Bronchitol’s safety and efficacy in Cystic Fibrosis patients aged 6 years and over, with a negative result on ...

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 ...

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 ...

NICE now recommends Colobreathe (Forest Labs)for Cystic Fibrosis infections.

NICE now recommends Colobreathe(colistimethate dry powder for inhalation) from Forest Labs as an option for treating chronic pulmonary infection caused by Pseudomonas aeruginosa in people with Cystic Fibrosis.

Colobreathe is recommended for those patients who would clinically benefit from continued Colobreathe treatment, but do not tolerate it in its nebulised form and the positive recommendation is subject to ...

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 ...

CHMP recommends bosutinib (Pfizer) for PH+CML

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the conditional marketing authorization of bosutinib from Pfizer, in the European Union (EU), 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) ...

Cinryze(ViroPharma) success in paediatric Hereditary Angio-Oedema

New data demonstrates that use of Cinryze (C1 esterase inhibitor [human]), from Viro Pharma, in pediatric patients provided relief from symptoms of Hereditary Angio-Oedema (HAO) attacks and reduced the rate of attacks. In the study, children had a nearly twofold reduction in number of HAE attacks while receiving Cinryze for prophylaxis compared with the time period during which they received ...

EMA rejects Raxone/Catena (Santhera) as a treatment for Leber's Hereditary Optic Neuropathy

Santhera Pharmaceuticals has announced that it has received a negative opinion on its Marketing Authorization Application (MAA) for Raxone (idebenone) as a potential therapy for Leber's Hereditary Optic Neuropathy (LHON). The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has notified Santhera that a narrow majority of CHMP members deemed Raxone not approvable at this time. Santhera ...

Votubia/Afinitor success in treatment of Renal Angiomyolipoma reported in The Lancet

Data published in The Lancet showed that patients on Afinitor (everolimus) tablets from Novartis, with non-cancerous kidney tumors known as Renal Angiomyolipomas associated with Tuberous Sclerosis Complex (TSC) experienced a significant reduction in tumor size and the absence of tumor progression. Additionally, a recent issue of The Lancet featured results from a separate everolimus trial demonstrating a reduction in ...

FDA approves Kineret(SOBI) for NOMID

Sobi- Swedish Orphan has announced that the FDA has approved Kineret (anakinra) for the treatment of children and adults with Neonatal-Onset Multisystem Inflammatory Disease (NOMID).

Kineret is the first and only FDA-approved therapy for NOMID, the most severe form of cryopyrin associated periodic syndromes (CAPS). This is the first approval allowing the use of Kineret in children. Kineret was ...

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 ...

Phase II success for AT 2220 in Pompe Disease

Amicus Therapeutics has announced positive preliminary results from all 4 dose cohorts in a Phase II study (Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT 2220 (duvoglustat HCl) co-administered with enzyme replacement therapy (ERT) for Pompe disease (Myozyme and Lumizyme). Myozyme and Lumizyme (alglucosidase alfa, or recombinant human GAA enzyme, rhGAA) are ...

Dexpramipexole (Biogen Idec) fails Phase III EMPOWER trial in ALS

Biogen Idec has reported top-line results of EMPOWER, a Phase III trial investigating dexpramipexole in people with Amyotrophic Lateral Sclerosis (ALS). The trial did not meet its primary endpoint, a joint rank analysis of function and survival, and no efficacy was seen in the individual components of function or survival. The trial also failed to show efficacy in its key ...

FDA will review Procysbi (Raptor Pharma) in April 2013

Raptor Pharmaceutical Corp. announced that the FDA will require additional time to complete its review of the New Drug Application (NDA) for RP103 (Procysbi), for the potential treatment of Nephropathic Cystinosis. In a notice received from the FDA, the initial Prescription Drug User Fee Act (PDUFA) goal date has been extended from January 30, 2013 to April 30, 2013. The ...

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 ...

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 ...

Migalastat (Amicus/GSK) disappoints in Phase III trial for Fabry Disease

Amicus Therapeutics and GSK announced the 6-month primary treatment period results from the first Phase III global registration study (Study 011) of investigational oral migalastat HCl monotherapy in males and females with Fabry Disease who had genetic mutations identified as amenable to migalastat HCl in a cell-based assay.

GL-3 clearance from the kidney interstitial capillaries is used as a ...

Early study shows Dantrium (JHP Pharmaceuticals) has promise for treating Duchenne Muscular Dystrophy

Scientists at UCLA have found Dantrium (sodium dantrolene), from JHP Pharmaceuticals, provides a powerful boost to drug therapy for Duchenne Muscular Dystrophy. The hope is that when used in combination, the drug will provide a one-two punch that will overcome the genetic mutations that cause Duchenne Muscular Dystrophy, and restore a missing protein needed for proper muscle function, and ...

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....

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 ...

PTC Therapeutics submits PTC 124 to the EMA for treatment of patients with Duchenne Muscular Dystrophy

A Marketing Authorization Application to the EMA seeking conditional approval for PTC 124 (ataluren), from PTC Therapeutics, for the treatment of patients with nonsense mutation Duchenne Muscular Dystrophy (nmDMD) has been accepted. Validation of the MAA confirms that the submission is complete and begins the EMA's Committee for Human Medicinal Products' (CHMP) review process.

The submission is based on a ...

Sobi files Kineret at EMA for CAPS treatment

Sobi (Swedish Orphan Biovitrium)) has filed an application for an EU Marketing Authorization with the European Medicines Agency (EMA) for Kineret (anakinra) for the indication of Cryopyrin Associated Periodic Syndromes (CAPS). The filing is based on positive safety and efficacy outcome data from a long-term treatment study in children and adults with neonatal-onset multisystem inflammatory disease (NOMID), the most severe ...

ACZ 885 (Novartis) success in Phase II study for Familial Mediterranean Fever

In the Phase II Familial Mediterranean Fever study, 100% of patients (nine out of nine) achieved at least a 50% reduction in the frequency of disease attacks during three months of ACZ 885 treatment from Novartis. All nine patients in the trial had previously experienced at least one attack per month over three months before receiving ACZ 885, while using ...

ACZ 885 (Novartis) continues positively in Phase II trial for TRAPS

An ongoing Phase II, open-label, multicenter study is investigating the efficacy and safety of ACZ 885 ,from Novartis, in patients with active Tumor Necrosis Factor (TNF) Receptor-associated Periodic Syndrome (TRAPS).

Patients received ACZ 885 150 mg (or increased to 300 mg for those without complete or almost complete response by Day 8) every four weeks for four months. ...

FDA award orphan status to A 4250 (Albireo) for Primary Biliary Cirrhosis and PFIC

The FDA has awarded A 4250, from Albireo, orphan-drug designation for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)and Primary Biliary Cirrhosis (PBC). Orphan designation was granted based on an extensive preclinical data package showing that A4250 has a high potency and a minimal systemic exposure. In addition, substantial benefits were shown in an animal model of cholestasis; the predominant ...

European Commission rejects Uplyso( Pfizer/Protalix) for Gaucher Disease

The European Commission has rejected Uplyso (taliglucerase alfa ) for Gaucher Disease as a grant of approval would breach the Orphan drug status of Vpriv (velaglucerase alfa) from Shire which has 10 years exclusivity from August 2010....

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 ...

Positive results from Phase III trial of Ruconest (Santarus) in patients with Hereditary Angioedema

A pivotal Phase III clinical study to evaluate the safety and efficacy of the investigational drug Ruconest (recombinant human C1 esterase inhibitor), from Santarus, for the treatment of acute attacks of angioedema in patients with Hereditary Angioedema (HAE) met the primary endpoint of time to beginning of symptom relief. A statistically significant difference in the time to beginning of symptom ...

AMG 145 (Amgen) success in RUTHERFORD study for HeFH

Amgen has announced that treatment with AMG 145 in combination with statin therapy, with or without ezetimibe, resulted in a reduction in low density lipoprotein cholesterol (LDL-C), or "bad" cholesterol, by up to 56 percent in patients with heterozygous familial hypercholesterolemia (HeFH) in the Phase II RUTHERFORD study.

AMG 145 is an investigational fully human monoclonal antibody directed against PCSK9, ...

EU approves Votubia (Novartis)for Renal Angiomyolipoma

Novartis received approval from the European Commission (EC) for Votubia (everolimus) tablets for the treatment of adult patients with renal angiomyolipoma associated with tuberous sclerosis complex (TSC) who are at risk of complications (based on factors such as tumor size or presence of aneurysm, or presence of multiple or bilateral tumors) but who do not require immediate surgery. This ...

GALNS (BioMarin) success in Phase III study for MPS IVA

BioMarin Pharmaceutical Inc.has announced that the pivotal Phase III study of GALNS met the primary endpoint of change in six-minute walk distance compared with placebo at 24 weeks in subjects receiving weekly infusions of GALNS at the dose of 2 mg/kg (p=0.0174). MOR-004 was a randomized, double-blind, placebo-controlled study evaluating two doses of GALNS (BMN-110, N- acetylgalactosamine-6-sulfatase) for the treatment ...

NICE recommends Bronchitol (Pharmaxis) for treatment of Cystic Fibrosis

The National Institute for Health and Clinical Excellence (NICE)has issued a positive recommendation in its Final Appraisal Determination for Bronchitol (mannitol dry powder) from Pharmaxis, clearing the way for reimbursement by the National Health Service. Bronchitol is recommended for adults whose lung function is rapidly declining and who do not use inhaled mucolytics for certain reasons. Bronchitol was filed at ...

NICE does not recommend Colobreathe (Forest Labs) for treatment of Cystic Fibrosis infection

NICE does not recommend Colobreathe (colistimethate dry powder for inhalation)from Forest Labs as a cost effective option for treating chronic pulmonary infection caused by P. aeruginosa in people with Cystic Fibrosis. The Committee noted that there was no economic analysis pitting the product against the nebulised form of colistimethate, while results of its preferred economic analysis showed that Colobreathe was ...

NICE recommends Tobi Podhaler (Novartis) for treatment of Cystic Fibrosis

NICE has recommended the use of Tobi Podhaler (tobramycin inhalation powder) from Novartis for the treatment of pseudomonas lung infection in Cystic Fibrosis patients aged six years and above. Tobi Podhaler may only be used in cases where treatment with the nebulised form of the drug is considered appropriate, i.e. when patients cannot have or do not respond to treatment ...

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 ...

FDA approve Aptima HPV Assay (Hologic) for detecting Human Papillomavirus

The FDA has approved the Aptima HPV 16 18/45 Genotype Assay, from Hologic, for use on its Tigris instrument system. It is the first FDA-approved test for genotyping human papillomavirus (HPV) types 16, 18 and/or 45, which are associated with approximately 80% of all invasive Cervical Cancers worldwide. The test is specifically approved for use adjunctively with the Aptima HPV ...

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 ...

Eteplirsen (Sarepta Therapeutics)success in extension trial for Duchenne Muscular Dystrophy

Sarepta Therapeutics has announced that treatment with its lead exon-skipping compound, eteplirsen, met the primary efficacy endpoint, increase in novel dystrophin, and achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT) over the placebo/delayed treatment cohort in a Phase IIb extension trial in Duchenne Muscular Dystrophy (DMD patients.

Eteplirsen administered once weekly at either ...

Phase III success for eliglustat tartrate (Sanofi/Genzyme) in Gaucher Disease

ENGAGE, the first Phase III trial of the investigational oral therapy, eliglustat tartrate,from Sanofi/Genzyme, in previously untreated patients with Gaucher disease type 1, met its primary endpoint. Patients treated with eliglustat tartrate had a statistically significant improvement in spleen size at nine months, compared with placebo. Spleen volumes in eliglustat tartrate treated patients decreased from baseline by a mean of ...

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 ...

First clinical study using lonafarnib (Merck Inc.) for treatment of Progeria

Results of the first-ever clinical drug trial using lonafarnib from Merck Inc., for children with Progeria, demonstrate the efficacy of a farnesyltransferase inhibitor (FTI), a drug originally developed to treat cancer. Twenty-eight children from sixteen countries participated in the two-and-a-half year drug trial, representing 75 percent of known Progeria cases worldwide at the time the trial began. Of those, 26 ...

CHMP recommends Votubia (Novartis) for Renal Angiomyolipoma

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for Votubia (everolimus) tablets for the treatment of adult patients with renal angiomyolipoma associated with tuberous sclerosis complex (TSC) who are at risk of complications (based on factors such as tumor size or presence of aneurysm, or presence of multiple or ...

Positive results from study of STX 209 (Seaside Therapeutics) for patients with Fragile X Syndrome

A new study shows STX 209 (arbaclofen), from Seaside Therapeutics, is effective for addressing the social withdrawal and challenging behaviors characteristic of Fragile X Syndrome, making it the first such discovery and potentially the first for autism spectrum disorder. The Phase II clinical trial initially recruited 63 subjects at 12 sites across the US and participants ranged in age from ...

EPI 743 (Edison Pharmaceuticals) granted orphan status for Leigh Syndrome in EU

Edison Pharmaceuticals announced today that the Committee for Orphan Medicinal Products at the European Medicines Agency, has granted orphan designation to EPI 743 for the treatment of Leigh syndrome. Leigh Syndrome is a very rare and severe disease caused by mutations in mitochondrial respiratory enzymes, leading mainly to neurological deficits and a poor survival for these patients. In June 2012 ...

FDA votes to support inhaled powder form of tobramycin (Novartis) for patients with Cystic Fibrosis

An FDA drug advisory committee has voted 13 to 1 that there was adequate evidence of efficacy and safety to support the use of tobramycin inhalation powder (TIP), from Novartis, for the management of Cystic Fibrosis (CF) patients whose lungs contain bacteria called Pseudomonas aeruginosa (Pa). It based its recommendation on three Phase III clinical studies involving more than 650 ...

Phase III data on Kalydeco (Vertex) shows benefits for Cystic Fibrosis patients

A Phase III study of Kalydeco (ivacaftor), from Vertex, for patients with Cystic Fibrosis shows the drug significantly improves lung function and reduces pulmonary exacerbations. The STRIVE study randomised 161 patients aged 12 and over with at least one copy of the G551D mutation in the CFTR gene to ivacaftor (150mg every 12 hours) or placebo. Results showed a mean ...

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 ...

Study shows Cinryze (Viro Pharma) effective for long-term treatment of Hereditary Angio-oedema

New results demonstrate that routine prophylactic use of Cinryze (C1 esterase inhibitor), from Viro Pharma, provided effectiveness and maintained a generally favorable safety profile in the majority of treated subjects with Hereditary Angio-oedema. Treated with Cinryze 1000 units every 3 to 7 days, subjects experienced a 93.7 percent reduction in attacks while taking prophylactic Cinryze (median of 0.19 attacks per ...

Sarepta Therapeutics success with eteplirsen in Phase IIb trial for Duchenne Muscular Dystrophy

Sarepta Therapeutics (formerly AVI Pharma), the developer of innovative RNA-based therapeutics, announced that treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT), over a placebo/delayed treatment cohort in a Phase IIb trial in Duchenne Muscular Dystrophy (DMD) patients. Eteplirsen administered once weekly at 50mg/kg over 36 weeks ...

Namenda (Merz) positive in Down's Syndrome trial

Researchers have found that Namenda (memantine)from Merz boosts memory function in those with Down's Syndrome, a major milestone in the treatment of this genetic disorder that could significantly improve quality of life. They studied 38 adolescents and young adults with Down's Syndrome. Half took the drug memantine, used to treat Alzheimer's disease, and the others took a placebo. Participants ...

CHMP recommends Glybera (uniQure) gene therapy for LPLD

uniQure announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion that recommends marketing authorization of Glybera (alipogene tiparvovec) as a treatment for Lipoprotein Lipase Deficiency (LPLD) under exceptional circumstances. LPLD is a very rare, inherited disease. Patients with the disease are unable to handle fat particles in their blood plasma, ...

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 ...

Positive results in Phase III study of VPRIV (Shire) for Gaucher-related bone disease

New data shows VPRIV (velaglucerase alfa for injection), from Shire, significantly improved selected markers of Gaucher-related bone disease in patients. A head-to-head Phase III study (HGT-GCB-039) of VPRIV and Cerezyme, and follow-on extension trial (HGT-GCB-044) of VPRIV, demonstrate a statistically significant improvement in lumbar spine (LS) bone mineral density in Gaucher patients starting at nine months of treatment with VPRIV, ...

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 refuses to recommend approval to Uplyso(Pfizer)for Gaucher Disease

The Committee for Medicinal Products for Human Use (CHMP) will not recommend approval to Uplyso (taliglucerase alfa) from Protalix/Pfizer for long-term enzyme replacement therapy for adults with Type 1 Gaucher disease because the drug works in the same way as Vpriv (velaglucerase alfa) from Shire and as Vpriv is an orphan drug it has 10 years exclusivity...

FDA issues Complete Response Letter for tafamidis (Pfizer) for TTR-FAP

The FDA has issued a Complete Response Letter for the transthyretin familial amyloid polyneuropathy drug, tafamidis from Pfizer. The FDA has requested the completion of a second efficacy study to establish substantial evidence of effectiveness prior to an approval. Tafamidis was approved in the EU as an orphan drug with the name Vyndaqel....

ACZ 885 (Novartis) success in Phase II trial for TRAPS

In a Phase II study, 90% of TRAPS patients treated with ACZ 885 (canakinumab) from Novartis, experienced clinical remission after only one week of treatment. Clinical remission included a clinically significant improvement of disease symptoms, as assessed by the treating physician. After two weeks of treatment, 95% of patients with TRAPS treated with ACZ885 had achieved a complete or almost ...

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 ...

FDA Advisory Committee votes on tafamidis (Pfizer) for TTR-FAP

The FDA Peripheral and Central Nervous System Drugs Advisory Committee voted on the clinical data package for tafamidis meglumine submitted by Pfizer. Tafamidis is a novel, investigational, oral therapy for the treatment of Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) in adult patients with symptomatic polyneuropathy to delay neurologic impairment. The Advisory Committee did not find substantial evidence of efficacy on a ...

FDA aproves Elelyso (Pfizer) for Type 1 Gaucher Disease

The FDA on 1st May 2012 approved Elelyso (taliglucerase alfa) from Pfizer for long-term enzyme replacement therapy to treat a form of Gaucher Disease. Gaucher Disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. The enzyme deficiency causes fatty materials (lipids) to collect in the spleen, liver, kidneys, and other organs. Elelyso is an injection ...

FDA approves Afinitor for treatment of Renal Angiomyolipomas

The FDA has on 26 April 2012 approved Afinitor (everolimus) tablets from Novartis for the treatment of adult patients with kidney tumors known as Renal Angiomyolipomas and Tuberous Sclerosis Complex (TSC), who do not require immediate surgery. This marks the first approval of a medical treatment in this patient population. The accelerated approval was based on the Phase III EXIST-2 ...

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....

IQWiG finds limited effect of Vyndaqel (Pfizer)in TTR-FAP

The German Institute for Quality and Efficiency in Health Care (IQWiG) has assessed Vyndaqel (tafamidis)from Pfizer at the request of the Federal Joint Committee. Only one of the two relevant studies (Fx-005) provided reliable data - and only for patients with a particular genetic defect (Val30Met). The second study (Fx1A-201) in adults with different genetic defects was not controlled, i.e. ...

Eteplirsen (AVI Biopharma) results in Phase IIb study in Duchenne Muscular Dystrophy

Treatment with eteplirsen from AVI Biopharma met the primary efficacy endpoint in a randomized, double-blind, placebo-controlled Phase IIb study in boys with Duchenne muscular dystrophy (DMD). Eteplirsen administered once weekly at 30mg/kg over 24 weeks resulted in a statistically significant (p ? 0.002) increase in novel dystrophin (22.5% dystrophin-positive fibers as a percentage of normal) compared to no increase in ...

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 ...

Pycnogenol (Horpagh Research) improves Menopausal transition

Pycnogenol extract from Horpagh Research Ltd, can ease Menopause and digestive problems according to a recent study. Seventy peri-menopausal randomly assigned women received either placebo or pine bark extract (100mg of Pycnogenol) daily for 8 weeks. The pine bark extract group reported a reduction in symptoms of Menopause including hot flushes, night sweats, mood swings, irregular periods,loss of libido and ...

FDA approves Gintuit (Organogenesis) for Mucogingival Conditions

The FDA on 9 March 2012 approved Gintuit from Organogenesis Inc., being the first cell-based product made from allogeneic human cells (from a donor unrelated to the patient) and bovine collagen. Gintuit is indicated for topical (non-submerged) application to a surgically created vascular wound bed in the treatment of mucogingival conditions in adults. With Gintuit, Organogenesis hopes to address the ...

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 ...

Procoralan (Servier) approved by European Commission for Chronic Heart Failure

Procoralan (ivabradine) from Servier Laboratories, the first selective If channel inhibitor, has been approved by the European Commission for the treatment of patients with Chronic Heart Failure. The European Commission´s decision to authorise this new indication for Procoralan followed the review of data from the SHIfT trial, the largest-ever morbi-mortality study of treatments for Chronic Heart Failure involving more than ...

Renadyl (Kibow Biotech) in study for Kidney Failure patients

Interim data from an expanded clinical trial of Renadyl, a probiotic dietary supplement from Kibow Biotech, confirms positive results for safety and quality of life previously established in a pilot-scale study for Kidney Failure patients. The probiotic microbial strains in the product formulation are "Generally Recognized As Safe" as per US FDA regulations. Renadyl, originally marketed under the name of ...

FDA clears Phase III trial of POL-103A (Polynoma) Melanoma vaccine

The FDA has granted clearance for Polynoma's Investigational new drug (IND) application for POL-103A, allowing it to proceed with Phase III clinical testing of this Malignant Melanoma vaccine. Using a combination of antigens from three proprietary melanoma cell lines, the POL-103A vaccine is intended to stimulate the body's immune system to fight the cancer. Preparation for the Phase III clinical ...

Positive data of subcutaneous Cinryze (Viro Pharma) with Enhanze (Halozyme) delivery platform for HAE

Results of a Phase II subcutaneous trial of Cinryze (C1 esterase inhibitor), from Viro Pharma, in combination with Halozyme's Enhanze technology, a proprietary drug delivery platform using Halozyme's recombinant human hyaluronidase enzyme (rHuPH20), showed positive results in patients with Hereditary Angio-oedema (HAE). This open-label, multiple-dose Phase II study was conducted in 12 subjects with HAE who previously participated in the ...

GSK files at FDA for H5N1 subtype Influenza vaccine

GSK has submitted a regulatory application to the FDA seeking approval for an H5N1 influenza vaccine. The Biologics License Application seeks approval for the “active immunisation for the prevention of disease in persons 18 years of age and older at increased risk of exposure to the influenza A virus H5N1 subtype contained in the vaccine” for use as directed by ...

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 ...

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....

FDA accepts new drug application from Pfizer for tafamidis for TTR-FAP

After a Refusal to File notification from the FDA for tafamidis from Pfizer, the FDA has now accepted the new drug application for Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP) a rare, progressive fatal neurodegenerative disease, which affects about 8,000 patients worldwide.The drug is approved as Vyndaqel in the EU....

FDA extends Berinert (CSL Behring) indication to self administration in HAE attacks

The FDA has approved an extended indication to allow self administration of Berinert (C1 inhibitor concentrate), from CSL Behring, in cases of acute abdominal or facial attacks of Hereditary Angioedema (HAE). This means that there is no need with Berinert for a patient to visit a clinic or hospital to receive the drug intravenously and allows the possibility of quicker ...

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 ...

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 ...

FDA extends review date of Uplyso (Pfizer)for Gauchers Disease until 1 May 2012

The FDA has extended its review by three months of the new drug application for Uplyso (taliglucerase alfa) from Pfizer/Protalix Biotherapeutics for the treatment of Gaucher's Disease .The effect delays the Prescription Drug User Fee Act action date until 1 May 2012. No additional data was requested by the FDA....

Vyndaqel(Pfizer) is EU approved for TTR-FAP

Vyndaqel (tafamidis), an orphan medicine from Pfizer to treat transthyretin familial amyloidosis in adult patients with symptomatic polyneuropathy (TTR-FAP), a neurogenerative disease, is EU approved The approval is based on studies that demonstrated the novel specific transthyretin stabiliser showed efficacy in delaying peripheral neurologic impairment, plus 51%-81% less deterioration in neurologic function, plus large and small fibre function compared with ...

Kalbitor (Dyax/Defiante Farma) is withdrawn from EMA for acute Hereditary Angioedema

Kalbitor from Dyax Corporation and Defiante Farmaceutica S.A. is withdrawn from its application to the EMA for the treatment of acute attacks of Hereditary Angioedema in patients 16 years and older. The CHMP had required further data. The drug is approved for the indication in the USA....

Huntexil (Neurosearch) trial suggests it is well tolerated and may offer benefits for patients with Huntington's Disease

A new study suggests that Huntexil (pridopidine), from Neurosearch, is well tolerated and warrants further study in patients with Huntington's disease (HD). Until now, no drug has been shown to improve the loss of the ability to move muscles voluntarily, and the only drug currently approved for HD, tetrabenazine, treats only chorea (involuntary movements) and is associated with serious side-effects. ...

CHMP rejects Glybera for Lipoprotein Lipase Deficiency

The Committee for Medicinal Products for Human Use (CHMP) has refused, a second time, to approve the gene therapy product Glybera (alipogene tiparvovec)from Amsterdam Molecular Therapeutics' for patients with lipoprotein lipase deficiency. The CHMP considered that there was insufficient evidence that its benefits outweigh its potential risks....

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 ...

Afinitor success in phase III study with angiomyolipomas, associated with Tuberous Sclerosis Complex

A Phase III study of Afinitor (everolimus)tablets from Novartus in patients with non-cancerous kidney tumors, or angiomyolipomas, associated with tuberous sclerosis complex met its primary endpoint of best overall angiomyolipoma response rate, which includes reduction in kidney tumor size and absence of new tumors. Findings from the trial, known as EXIST-2, were presented today at the International TSC Research Conference ...

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 ...