An expensive rare disease drug was approved by the US Food & Drug Administration in September despite findings by eight 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 FDA 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 eight reviewers recommended against approval.
For years, the US-based company has been seeking approval for elamipretide to treat the life-threatening mitochondrial disorder that affects about 150 people in the country, mostly males. The disease typically presents as severe heart failure in infancy and often leads to premature death.
The FDA declined to consider elamipretide for full approval in 2021 and rejected the company’s application in May 2025 before suggesting Stealth apply for an expedited process. It approved the drug just more than a month after Stealth refiled its application.
Stealth CEO Reenie McCarthy said the FDA made an informed, science-based decision to approve Forzinity, recognising both the strength of the data and the urgent unmet need in Barth syndrome.
The Trump administration has made rare disease drug approvals a priority, and had just introduced a new policy that allows applications to proceed based on one strong study and other confirmatory evidence.
FDA drug approvals are not typically contentious among evaluation teams, according to experts and a Reuters review of approval documents from 2024, although agency chiefs have used their authority in the past to push through approvals.
Shortcomings
Documents show that FDA Office Chief Hylton Joffe signed off on the accelerated approval, citing a 10-person, 192-week study in which the drug boosted knee strength by more than 45%, as well as the disease’s rarity and severity.
But reviewers listed various concerns, including that the drug failed to beat a placebo in a 28-week, late-stage study, and that the non-placebo study Joffe cited failed to show a direct link between the treatment and improvements.
Placebo patients improved almost as much as those on Forzinity in a six-minute walk test – the study’s main measure. It also failed to show significant gains on secondary goals like sit-to-stand tests, reviewers said.
FDA clinical team leader Charu Gandotra recommended against approval to Joffe, having argued in May that Stealth’s data did not “provide substantial evidence of effectiveness to support traditional or accelerated approval”.
Ten reviewers did sign off on the approval, documents show.
The US Department of Health and Human Services, which oversees the FDA, said additional reviewers did not sign the final Forzinity approval memo because their concerns were resolved earlier or addressed in other review documents. HHS declined to share those documents.
Joffe and Gandotra did not respond to requests for comment.
Reuters article – FDA clears costly rare disease drug despite objections (Open access)
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