The Food and Drug Administration was in dilemma for a long time over a potential new drug meant to reduce the progression of Duchenne muscular dystrophy, a chronic disease affecting boys leaving them with wheelchairs in their teens. Many patients die from the disease in their 20s.
On Monday, Massachusetts-based Sarepta Therapeutics informed that the FDA has detailed a priority review path for the drug, eteplirsen, and also indicated a willingness to consider for its early approval. This will allow the company to file fresh approval application with the FDA.
Chris Garabedian, Sarepta's Chief Executive, said, "This provides the opportunity to get the drug approved and in the hands of all the boys who can benefit from it sometime in 2015". Such was not the scene about five months ago when the FDA said to Sarepta that it would be too early for the company to seek approval.
In its initial trial involving 12 boys, the drug showed growth and halted physical decline. It somewhere brought hope among many parents whose were seeking for a treatment. To add pressure on the FDA, parents and families carried out campaigns, hired a public relations firm and they were all over social media and YouTube.
They simply argued that it is on the FDA whether their sons would be the last generation of boys to die from Duchenne or the first to survive. A petition of more than 100,000 signatures for White House was also presented.
It now seems that all these actions have resulted in at least a consideration by the FDA's side. But the FDA has not guaranteed the approval to the drug for the treatment that affects around 15,000 boys in the United States.
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