The Food and Drug Administration is putting off a decision on whether to approve a controversial drug that patient advocates and some senators say is the only hope for children with a fatal rare disease.
Sarepta Therapeutics said Wednesday that the agency wouldn’t make a decision as expected on Thursday on the drug eteplirsen to treat Duchenne Muscular Dystrophy, a rare disease that mainly targets young boys. The decision comes as hundreds of lawmakers have pushed the agency to approve the treatment because there is no other for Duchenne.
The agency had set a goal to decide on the drug on Thursday. However, Sarepta said the FDA needs more time.
The agency did not give a new date but told Sarepta that it will “strive to complete their work in as timely a manner as possible.”
A panel of experts recently found that the drug wasn’t effective and recommended against approval. The FDA does not have to abide by the recommendations of its experts but it usually does.
The decision comes as the agency takes heat from lawmakers to approve the drug. Two Republican senators wrote to the FDA on Friday asking it to approve the drug. In February, more than 100 House lawmakers pushed for expedited approval of it as well.
The letters of support are in response to a concerted grassroots effort by patient advocates to get the agency to approve the product.
