News

FDA approves Ilaris (Novartis) for treatment of Systemic Juvenile Idiopathic Arthritis

Novartis announced that the FDA has on 10 May 2013 approved Ilaris (canakinumab) for the treatment of active Systemic Juvenile Idiopathic Arthritis (SJIA) in patients aged 2 years and older. Ilaris is the first interleukin-1 beta (IL-1 beta) inhibitor approved for SJIA and the only treatment approved specifically for SJIA that is given as a once-monthly subcutaneous injection.

SJIA ...

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 Vyvanse (Shire) as maintenance treatment of ADHD for children and adolescents

The FDA has on 1 May 2013, approved the prescription medication Vyvanse (lisdexamfetamine dimesylate) Capsules, (CII) from Shire ,as a maintenance treatment in children and adolescents with Attention-Deficit/Hyperactivity Disorder (ADHD). Vyvanse is currently approved as a maintenance treatment in adults with ADHD. With this new approval, Vyvanse becomes the only stimulant approved for maintenance treatment in children, adolescents, and ...

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 ...

European Commission approves Hexyon/Hexcima (Sanofi Pasteur) Six in One vaccine

Sanofi Pasteur, the vaccines division of Sanofi has announced that the European Commission, on 22 April 2013, approved Sanofi Pasteur’s 6-in-1 pediatric vaccine Hexyon/Hexacima (DTaP-IPV-Hib-HepB vaccine) for primary and booster vaccination of infants from six weeks of age. Hexyon/Hexacima is the only fully liquid, ready-to-use, 6-in-1 vaccine to protect infants against diphtheria, tetanus, pertussis (whooping cough), Hepatitis B, poliomyelitis ...

Positive topline results from Phase III trial of Enterovirus 71 vaccine (Sinovac Biotech) against Hand, Foot and Mouth Disease

Preliminary top-line data from a Phase III clinical trial assessing the efficacy, immunogenicity and safety of Enterovirus 71 vaccine, from Sinovac Biotech, against Hand, Foot and Mouth Disease, showed that the vaccine was 95.4% efficacious against HFMD. The Phase III trial involving 10,000 healthy infants of 6 to 35 months old showed good immunogenicity and safety.

The overall incidence of ...

FDA halts pediatric trials of Sensipar(Amgen) for Hyperparathyroidism

The FDA has stopped all pediatric clinical trials of Sensipar (cinacalcet hydrochloride) from Amgen, after the recent death of a 14-year-old patient in a trial. The FDA continues to gather information on the circumstances surrounding the patient’s death. Sensipar is a medication used to decrease the release of parathyroid hormone (PTH) from the parathyroid gland. Sensipar lowers high PTH levels ...

CHMP recommends Hexyon/Hexacima vaccine (Sanofi Pasteur)

The CHMP has recommended market approval for the 6-in-1 pediatric vaccine Hexyon/Hexacima (DTaP-IPV-Hib-HepB vaccine), from Sanofi Pasteur. The new vaccine will be commercialized under the brand name Hexyon in Western European countries by Sanofi Pasteur MSD, the joint venture between MSD and Sanofi Pasteur, and under the brand name Hexacima in Eastern European countries by Sanofi Pasteur. The new vaccine ...

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 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 ...

CHMP recommends Pegasys(Roche) for paediatric use in Hepatitis C

The CHMP recommends Pegasys (peginterferon alfa-2a) from Roche, in combination with ribavirin, be indicated for the treatment of chronic Hepatitis C in treatment-naïve children and adolescents five years of age and older, who are positive for serum hepatitis-C-virus (HCV) RNA. When deciding to initiate treatment in childhood, it is important to consider growth inhibition induced by combination therapy. The ...

CHMP recommends extension of indication of Humira(AbbVie) in JIA

The CHMP recommends that Humira (adalimumab) from AbbVie, in combination with methotrexate is to be indicated for the treatment of active Polyarticular Juvenile Idiopathic Arthritis, in children and adolescents aged 2 to 17 years who have had an inadequate response to one or more disease-modifying anti-rheumatic drugs (DMARDs). Humira can be given as monotherapy in case of intolerance to ...

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 VariZIG (Cangene Corpn) for Chicken Pox treatment

The FDA on 21 December 2012 approved VariZIG,a varicella zoster immune globulin preparation from Cangene Corporation, for reducing the severity of Chicken Pox (varicella zoster virus) infections in high risk individuals when given within four days after exposure. Varicella zoster immune globulin (VZIG) has been shown to lower the risk of severe infections if given soon enough after exposure. An ...

Results of Phase III studies show substantial benefits of ACZ 885 (Novartis) in patients with Juvenile Idiopathic Arthritis

Results of two Phase III trials show ACZ 885 (canakinumab), from Novartis, provided substantial symptom relief in young patients with systemic Juvenile Idiopathic Arthritis (SJIA). In addition, ACZ 885 delayed disease flare recurrence and allowed patients to substantially reduce or discontinue use of corticosteroids.

In beta-SPECIFIC 1 (trial-1), 84% of SJIA patients treated with ACZ 885 experienced at least a ...

Antares Pharma files Otrexup at FDA for Rheumatoid Arthritis

Antares Pharma has submitted a New Drug Application (NDA) to the FDA for Otrexup, a combination product for the delivery of methotrexate (MTX) using Medi-Jet technology. Otrexup was developed for easy subcutaneous administration of MTX to enhance the treatment of rheumatoid arthritis (RA), poly-articular-course juvenile RA and moderate to severe psoriasis....

Positive European regulatory procedure for Elvanse (Shire) for ADHD

Shire plc announced a positive outcome from the European Decentralised Procedure (DCP) for Elvanse (to be known as Tyvense in Ireland). Elvanse is indicated as part of a comprehensive treatment programme for Attention Deficit/Hyperactivity Disorder (ADHD) in children aged 6 years of age and over when response to previous methylphenidate treatment is considered clinically inadequate. The MHRA of the UK, ...

RTS.S anti malaria vaccine further results in African study

Results from a pivotal, large-scale Phase III trial, published online in the New England Journal of Medicine, show that the RTS,S malaria vaccine candidate can help protect African infants against malaria. When compared to immunization with a control vaccine, infants (aged 6-12 weeks at first vaccination) vaccinated with RTS,S had one-third fewer episodes of both clinical and severe malaria and ...

FDA approves Affinity Pixie Oxygenation System (Medtronic Inc) for use in Heart Surgery on children

The FDA have given 510(k) clearance for the Affinity Pixie Oxygenation System, from Medtronic Inc, for use during lifesaving open-heart (cardiopulmonary bypass) surgeries in neonates, infants and small children, including those with congenital heart defects. The Affinity Pixie Oxygenation System serves as a child’s lungs during open-heart surgery by removing carbon dioxide and adding oxygen to the child’s blood before ...

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 ...

FDA approves Quillivant XR (Next Wave) for ADHD

The FDA has approved Quillivant XR (methylphenidate hydrochloride) from Next Wave Pharma for extended-release oral suspension for treatment of Attention Deficit Hyperactivity Disorder (ADHD). Quillivant XR is the first once-daily, extended-release liquid methylphenidate available for patients with ADHD.The drug will be launched early in 2013....

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 ...

TC 5619 (Targacept) fails Phase II trial in ADHDi

Targacept Inc., has announced top-line results from a Phase II trial of TC-5619 as a treatment for inattentive-predominant attention deficit/hyperactivity disorder (ADHDi). In the trial, TC-5619 did not meet the primary outcome measure, change from baseline on the inattention subscale of the Conners' Adult ADHD Rating Scale-Investigator-Rated (CAARS-INV), after four weeks of treatment versus placebo. Across the study measures, patients ...

FDA reviews sNDA of Vyvanse (Shire) as maintenenace treatment for young patients with ADHD

The FDA has accepted the filing for review of a supplemental New Drug Application (sNDA) for Vyvanse (lisdexamfetamine dimesylate) Capsules, from Shire, as a maintenance treatment in children and adolescents ages 6 to 17 years with Attention-Deficit/Hyperactivity Disorder (ADHD). This application is based on results of a Phase IIIb, randomized withdrawal, multicenter, extension study to evaluate the long-term maintenance of ...

FDA approves Afinitor Disperz(Novartis) for SEGA Tumours

The FDA has on 29 August 2012 approved Afinitor Disperz (everolimus tablets for oral suspension; Novartis), a new pediatric dosage form of Afinitor (everolimus) indicated for treatment of Tuberous Sclerosis Complex (TSC) in patients aged >1 year who are diagnosed with Subependymal Giant Cell Astrocytoma (SEGA) that cannot be treated with surgery. Afinitor Disperz is the first approved pediatric-specific dosage ...

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 ...

Phase III trials shows Vyvanse (Shire) effective in children and adolescents with ADHD

Results from a Phase III extension study of Vyvanse (lisdexamfetamine dimesylate), from Shire, show that after 6 months of treatment, a significantly lower proportion of subjects with ADHD experience treatment failure compared to those on placebo. This study was designed to evaluate the long-term efficacy and safety of Vyvanse for the treatment of ADHD in children and adolescents aged 6 ...

NICE endorses Victrelis (Merck Inc.)for Hepatitis C genotype 1 infection

In its final draft guidance, NICE recommends Victrelis (boceprevir) from Merck Inc., in combination with Roche’s Pegasus (peginterferon alfa) and Copegus (ribavirin) injections,as an option for the treatment of genotype 1 Chronic Hepatitis C in adults with compensated liver disease.A final endorsement is expected next month. Hepatitis C has a poor diagnosis and low treatment compliance rate, with a high ...

Avastin (Roche) possible new treatment for Haemorrhagic Telangiectasia

A new study suggests that Avastin (bevacizumab), from Roche, may help to improve cardiac function and reduce the number of potentially life-threatening nose bleeds caused by a rare genetic vascular disorder known as Haemorrhagic Telangiectasia. In a Phase II study of 24 patients treated in France, the duration of nosebleeds dropped from a mean of 221 minutes per month to ...

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 ...

Repligen files Secreflo with EMA for detection of Pancreatic Duct Abnormalities

Repligen has submitted a marketing authorization application (MAA) for SecreFlo (synthetic human secretin) as an agent to improve the detection of pancreatic duct abnormalities in patients with Pancreatitis. SecreFlo stimulates secretion of watery fluid into the pancreatic ducts. When the ducts are filled with water they are more effectively visualised. FDA decision expected on 21 June 2012....

Shire initiates Phase IV trials to compare Vyvanse ands Concerta

Shire will initiate two Phase IV clinical trials to compare Vyvanse (lisdexamfetamine dimesylate) Capsules, (CII) to Concerta® (methylphenidate HCl) Extended-Release Tablets, (CII).e two Phase 4 clinical trials are randomized, double-blind, multi-center, parallel-group, active-controlled studies. They are designed to explore differences in efficacy between Vyvanse and Concerta in adolescents ages 13 to 17 with ADHD. This clinical trial program will utilize ...

Da Vinci Surgical System a benefit for HPV-related Oral Cancer

A study of robotic surgery conducted with the da Vinci Surgical System, and made through patients' mouths reveals it provides excellent results in removing squamous cell carcinoma at the back of the throat, especially in patients with HPV. The study followed 66 patients with Oropharyngeal Cancer who underwent transoral robotic surgery. Every few months, the patients had imaging studies, scans ...

CHMP recommends Remicade (Janssen Biologic) for Paediatric Ulcerative Colitis

The CHMP has issued a positive opinion for Remicade (infliximab) from Janssen Biologic for the treatment of severely active Ulcerative Colitis in paediatric patients aged between six to 17 who have had an inadequate response to conventional therapy, or who or intolerant to, or have medical contraindications to these therapies. The recommendation is based on a Phase III randomised, open-label ...

Intuniv (Shire) shows positive response for children with ADHD

A new trial has assessed the efficacy and safety of Intuniv (guanfacine extended release), from Shire, as an adjunct to psychostimulants in children and adolescents diagnosed with ADHD who had a suboptimal response to a psychostimulant alone. Participants continued their stable dose of psychostimulant given in the morning and were randomised to receive Intuniv in the morning, Intuniv in the ...

FDA finds ADHD drugs have no link to Cardiovascular problems

The US FDA has announced that a large, recently-completed study in children and young adults treated with medication for Attention-Deficit/Hyperactivity Disorder (ADHD) has not shown an association between use of certain ADHD medications and adverse cardiovascular events. These adverse cardiovascular events include stroke, heart attack (myocardial infarction or MI), and sudden cardiac death. The study was conducted with 1,200,438 children ...

Genes predict child's response to Equasym in treatment of ADHD

New research suggests that the genetic characteristics of a child with ADHD may offer a useful indicator in selecting the treatment most suitable for them. The study, published in the Journal of the American Academy of Child and Adolescent Psychiatry, found that out of 89 children aged from 7-11 with ADHD, those with specific variations of the dopamine receptor D4 ...

Vyvanse positive in European Phase III study for ADHD

Vyvanse- lisdexamfetamine (LDX) from Shire is the long acting, prodrug of dexamfetamine for the treatment of ADHD and is currently licensed only in the US, Canada and Brazil. A study, conducted at 48 sites across Europe, showed that LDX demonstrated efficacy on the primary and key secondary measures compared to placebo, and a safety profile consistent with the known effects ...

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 ...

Surfaxin is re-filed at FDA for Respiratory Distress Syndrome

The FDA has accepted the re-application for the humanised surfactant Surfaxin (lucinactant) from Discovery Laboratories as a preventive therapy for respiratory distress syndrome (RDS) in premature infants as complete and has classified it as a Class 2 submission, setting 6 March 2012 as the Prescription Drug User Fee Act action date. Respiratory Distress Syndrome is a condition in which premature ...

FDA approves Remicade for Pediatric Ulcerative Colitis

The FDA has approved Remicade (infliximab), from Janssen Biotech, for the treatment of moderately to severely active Ulcerative Colitis (UC) in pediatric patients who have had an inadequate response to conventional therapy. The approval was supported by evidence from studies of Remicade in adults with Ulcerative Colitis plus additional data from a pivotal Phase III trial evaluating the efficacy and ...

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 ...