Exclusive: US FDA cleared pricey rare disease drug over reviewer objections
NEW YORK, Nov 5 (Reuters) - The U.S. FDA approved a pricey rare disease drug in September despite findings by its data reviewers that the treatment, while safe, was no more effective than a placebo, a Reuters review of agency documents found.
The urgent need among patients, along with some signs of improved motor skills, helped drive the agency’s decision, the documents show.
The Food and Drug Administration on September 19 gave its backing to Stealth Biotherapeutics’ elamipretide, which will be sold as Forzinity and priced at up to nearly $800,000 a year. It will be the first treatment for Barth syndrome, although FDA documents show nearly a dozen reviewers recommended against approval.
To me, without understanding more details than the headlines, seeing “no more effective than placebo” and “costs over 800,000 dollars a year” it sounds like the FDA just approved people getting taken advantage of.
The Massachusetts-based company had for years been seeking approval for elamipretide to treat the life-threatening mitochondrial disorder that affects about 150 people in the U.S., mostly males. The disease typically presents as severe heart failure in infancy and often leads to premature death.
Perhaps the rodents are scurrying around beneath Secretary Kennedy.
In May 2025, when the FDA initially rejected Forzinity, HHS did not respond to media inquiries about the decision.
The FDA later reversed course and granted accelerated approval, citing muscle strength improvements and unmet medical need — but Kennedy’s name was absent from all official documents and public commentary
Haven’t read anything further yet… but sometimes this journey to Honest Medicine feels so tough, 2 steps forward and two back. Thanks for sharing @jrgerber