Sarepta Drug Eteplirsen to be Reviewed by FDA under Accelerated Approval Pathway
Sarepta Therapeutics announced that U.S. FDA has indicated alternate path to approval for its muscle disorder drug. The experimental drug to arrest the progression of Duchenne muscular dystrophy in young boys was not able to convince the FDA panel over the last few months. The latest news has raised fresh hope for the approval of the drug. Sarepta Therapeutics stock jumped nearly 64 percent in the stock market after the positive news.
Families of patients led an aggressive campaign to pressurize FDA for the drug approval, considering the better chances of Sarepta’s exon-skipping drug candidate compared to other treatment options. The muscle disorder pushed most of the patients to wheelchairs and to death in certain cases by age 20-30 years.
The parents had also started an online petition to the White House for the approval of the drug by FDA. Social media websites were also used to fuel the campaign for the drug approval by concerned parents.
Chris Garabedian, President and CEO of Sarepta Therapeutics said, “We also appreciate that the FDA shares our urgency in dosing a broader base of eteplirsen patients and has encouraged us to begin the clinical program with our follow-on exon-skipping drugs as soon as possible.”
Massachusetts-based Sarepta Therapeutics informed that they have positive results of a recently conducted study to support the impact of drug in duchenne muscular dystrophy patients. The company will file a new drug application with FDA. The accelerated approval pathway is offered by FDA for drugs which are for serious ailments, with no treatment options.
The company will carry out open-label, historically controlled study for the drug impact on young boys. Many concerned parents have shared that the drug offers benefits to their kids. Due to lower production of essential protein dystrophin, the muscle function in patients in impaired. The condition leaves most of the patients immobile by teenage. The disease also affects respiratory and heart muscles at later stage, causing serious health issues.
In United States, duchenne muscular dystrophy affects nearly 15,000 boys. As per CDC data, one in 3,600 boys suffers from the condition. Sarepta will file for approval by the end of the year. Sarepta drug named eteplirsen is expected to launch by 2016, subject to FDA approval. Earlier, the company was expecting the launch in 2018. The company expects marketing nod by mid 2015.
The latest move by FDA does not guarantee approval of the drug, but chances are higher. The investors picked up the stock after the positive news. The company announced that it will initiate additional eteplirsen clinical studies to confirm the positive impact of the drug. The company added that the medical team is working hard on similar drugs to treat boys with other exon deletions as well.
Prosensa Holding NV is another company working on DMD drug drisapersen. The experimental drug failed to better the plight of boys suffering DMD in a clinical trial that ended in September 2012. The stock price of Prosensa also jumped raising fresh hope for their experimental drug.
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