FDA's Controversial Approval: Forzinity Green-Lit Amid Criticism
The FDA approved Forzinity, a costly drug for Barth syndrome, despite its limited efficacy over a placebo. The decision faced internal opposition but was driven by patient demand and advocacy. Stealth Biotherapeutics plans to launch the drug in December, pricing it at nearly $800,000 annually.
The U.S. Food and Drug Administration has faced criticism after approving a high-priced drug for Barth syndrome, a rare disease, despite data suggesting it is no more effective than a placebo. According to a Reuters review, while the drug elamipretide, branded as Forzinity, was deemed safe, its efficacy remains disputed among FDA data reviewers.
Despite nearly a dozen reviewers advising against approval, the FDA gave the green light on September 19, following signs of improved motor skills in some patients and a significant need within the patient community. The drug, developed by Stealth Biotherapeutics, becomes the first treatment option for Barth syndrome, impacting around 150 individuals in the U.S., mainly males. The disease often results in severe heart failure in infancy.
Documents reveal Hylton Joffe, an FDA office chief, cited a study showing a 45% increase in knee strength among other factors for the accelerated approval, discounting some findings from previous trials. The approval process reportedly faced pressure from lawmakers, physicians, and patient advocates urging swift action. Stealth Biotherapeutics is preparing to launch Forzinity, priced at $800,000 annually, amid plans for further studies to confirm its benefits.
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