The US Food and Drug Administration has given an accelerated approval to the first drug of Sarepta Therapeutics Inc (NASDAQ:SRPT) that helps slow down further advancement in the disease, Duchenne muscular dystrophy.
Duchenne Muscular Dystrophy
More popularly known as DMD, Duchenne muscular dystrophy is one of the types of muscular dystrophy that is caused by the absence of a certain protein and helps keep muscles scatheless and unbroken. This protein is called dystrophin.
Duchenne muscular dystrophy is a genetic disorder redressed by an escalating muscle weakness and degeneration. There are nine types of muscular dystrophy and Duchenne muscular dystrophy is one of them.
Sarepta’sDuchenne Drug Granted with an Accelerated Approval by the FDA
The first and newest Duchenne Drug by known drug manufacturer, Sarepta Therapeutics Inc. has been granted an accelerated approval by the FDA. The US Food and drug administration took six months more from its primary deadline in executing its decision. The approval is still conditional since the basis was predominantly from a study conducted, which involved only 12 boys. A much numerous and diverse trial is already on its way.
The drug has been approved to cure patients who have a specific gene mutation. About 13 percent of patients share this specific gene mutation. The drug, more generically known to be Eteplirsen has been given and was revealed to have a brand name of Exondys51.
Janet Woodcock, the director of the Center for Drug Evaluation and Research of the FDA, has termed the development of drugs and rare disease “especially challenging”.
Janet Woodcock, the director of the Center for Drug Evaluation and Research of the FDA, has termed the development of drugs and rare disease “especially challenging”. She said in a statement that: “Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”
On an interview with the mother of the oldest boy who took part in the study of 12 boys, Terri Ellsworth said that right after the news broke out at 10 in the morning; she had already seen the news on twitter. For years, Ellsworth had been an advocate of the government’s approval for the said Duchenne drug. Also according to her, she sees this approval of this particular Duchenne medicine to propel other drug manufacturers to also develop their own brand for the treatment of Duchenne muscular dystrophy.