FDA grants accelerated approval to controversial muscular dystrophy drug

From The Washington Post:

After months of advocacy and speculation, the Food and Drug Administration today granted accelerated approval to the first treatment for a rare form of muscular dystrophy. The decision pitted the passionate testimony of patients and families against an FDA advisory committee that wasn’t convinced the drug worked.

The approval of Sarepta Therapeutics’ drug, eteplirsen, was a huge, emotionally fraught victory for families with Duchenne muscular dystrophy — one of the most vocal and involved patient communities since the days when HIV patients pushed the agency to approve more drugs. The approval specifies that the clinical benefit of the drug “has not been established” and is contingent on a follow-up clinical trial. The process has been closely watched by parents, patient advocates and biotech investors, and has been seen as an important test case for the effort to integrate the patients’ point of view more deeply into the approval process.

“Obviously it’s a big relief, and it may have come just in time to help save some of these young boy’s lives, which is so important,” said Christine McSherry, whose adult son Jett has been taking the drug in a safety study. “But it wasn’t timely enough.”

Duchenne muscular dystrophy is a devastating, invariably fatal disease that affects between 9,000 and 12,000 boys in the United States. Boys with the disease grow progressively weaker, becoming reliant on a wheelchair in their teens and dying in their 20s or 30s.

The news was cheered by parents and advocates, including Terri Ellsworth, whose said she was stunned, happy and relieved. Her 15-year-old son, Billy, has been participating in a clinical trial for the drug and hadn’t heard the news before he left for school. She planned to visit him later today to surprise him with …

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