Sarepta has become the first company to get US approval for a drug to treat Duchenne muscular dystrophy, with the FDA overruling its own advisors in backing the drug.
The biopharma company still has to conduct a new clinical trial to allay lingering concerns about its efficacy and if that fails, the approval will be rescinded.
In the meantime Sarepta has said it will move swiftly to launch the drug, which has been given a list price of $300,000 a year. [YIKES!]
"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," said Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research (CDER).
Documents published by the FDA reviewers ahead of an advisory committee meeting raised major doubts about its efficacy, and those concerns were played out at the meeting with only three of 10 panelists voting that the drug is effective in DMD (read “FDA Advisory Panel Deals a Blow to ‘Patient Power’ Re: Experimental DMD Drug”).
The panel meeting was inundated with patients and their families calling for the FDA to take a chance and green light the drug, and it seems that lobbying effort swayed the agency (read “Patient Advocates - and Docs with Ties to Pharma - Turn Up the Heat on FDA”).
The approval decision is far from unanimous however, with Woodcock in opposition to fellow CDER officials including CDER deputy director Ellis Unger who appealed the decision to approve via the FDA's internal disputes procedures. Meanwhile, the FDA's lead reviewer Ronald Farkas - who criticised the quality of the eteplirsen data in a recent report - left the agency a few weeks ago.