Interesting story about FDA dilemma - Sarepta have still not...

Interesting story about FDA dilemma - Sarepta have still not provided solid efficacy data across all of their approved treatments in the USA.

After all these years "Sarepta has yet to convert the accelerated approval to a full one..."

While approving Sarepta drugs has helped spur new investment in DMD, the FDA now needs to resolve the lack of progress in confirmatory trials...

Question: wonder if Dr Voit will measure dystrophin production in this Phase 2B trial, could the control /lowering of inflammation help with maintaining dystrophin levels?

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US spent $3B on DMD treatments as confirmatory studies still underway, JAMA paper says
Lia DeGroot

The US has so far doled out about $3 billion on three of Sarepta Therapeutics’ therapies for Duchenne muscular dystrophy, even as all three of the confirmatory trials have yet to read out, Harvard researchers found.
In the paper published Monday, the authors called on policymakers to ensure confirmatory trials are completed in a timely manner, and they suggest limiting Medicare and Medicaid reimbursement for accelerated approval drugs until clinical benefits are verified.

The study looked at Exondys 51 (eteplirsen), Vyondys 53 (golodirsen), and Amondys 45 (casimersen), noting that the majority of US spending, about $2.6 billion, was on eteplirsen, which won a contentious approval in 2016 after an advisory committee voted against it.

“As the FDA considers approving multimillion-dollar therapies with novel mechanisms of action and scant preapproval evidence of clinical benefit, policymakers should encourage timely completion of confirmatory trials and minimize financial risk in case treatments prove ineffective,” the researchers wrote.
The FDA in November 2023 published bioequivalence guidance for any generic drugmakers looking to compete with Exondys 51, even as Sarepta has yet to convert the accelerated approval to a full one. Sarepta previously said its key patents on Exondys 51 don’t expire until June 2025.

“I never thought that drug [eteplirsen] would be able to complete a confirmatory trial, but related exon skippers could, and show the surrogate,” former FDA Deputy Commissioner Janet Woodcock told Endpoints in an interview in January. “The question was the level of dystrophin produced. And it gets into the issue of biopsying children who are becoming progressively paralyzed. It wasn’t a very good measure.”
The researchers also urge policymakers to consider alternative payment contracts for cell and gene therapies. Sarepta did not immediately return Endpoints News’ request for comment.

The paper’s findings come as the FDA is pushing its accelerated approval pathway for gene therapies, with Peter Marks saying last month that it would become the “norm” for gene therapies moving forward.
But accelerated approvals haven’t come without controversy. Marks made the rare decision last year to override reviewers’ decision on Sarepta’s DMD therapy Elevidys, and it’s now on track for a label expansion this summer despite failing a confirmatory trial.