STAT+: A rare disease drug is rejected, even as the FDA talks about new approval pathway
Instead of approving Stealth BioTherapeutics' drug for Barth syndrome, the FDA withheld its endorsement, but decided it may be possible for Stealth to pursue accelerated approval.
For the past decade, Stealth BioTherapeutics has ridden a rollercoaster trying to convince the Food and Drug Administration to approve its ultra-rare disease drug. Now, the company has encountered yet another twist — an unexpected regulatory rejection that will not only delay access and strain its finances, but ensure some of the most vulnerable patients are denied the treatment.
At issue is a medication for Barth syndrome, a rare illness that causes an enlarged heart, muscle weakness and a shortened life expectancy. The disease afflicts up to 150 people in the U.S., an extremely small number that has, at times, made it difficult for the company and the FDA to find a way to generate enough of the right kind of study data to make the drug available to patients.
But after bouncing between different agency divisions and nearly ending its development efforts, Stealth last October won a key victory — a majority of panelists on an FDA advisory committee voted to recommend approval. The FDA, however, then missed a previously scheduled deadline this past January to complete its marketing review, pushing a decision to April. But the agency missed that deadline, too, suggesting the vast number of job cuts were a factor.
