FDA's Controversial Approval of $800,000 Rare Disease Drug Sparks Debate
In September, the U.S. FDA approved Stealth Biotherapeutics' elamipretide, branded as Forzinity, for Barth syndrome despite mixed reviews on its efficacy. Priced at $800,000 yearly, the decision was driven by pressing patient demands and mild improvements in motor skills. The approval process has sparked discussions on rare disease drug policies.
The U.S. Food and Drug Administration's recent decision to approve Stealth Biotherapeutics' elamipretide, marketed as Forzinity, has ignited a debate over drug approval processes for rare diseases. Despite concerns over its effectiveness compared to a placebo, the drug received approval amidst urgent demands from patients and proponents.
The treatment, intended for the rare Barth syndrome, marks the first of its kind and comes with a hefty price tag nearing $800,000 annually. This approval arrived after a tumultuous application history, with the FDA previously declining a full approval in 2021 before suggesting an expedited process.
Proponents of Forzinity, including patient advocacy groups and lawmakers, exerted significant pressure on the FDA to approve the drug. With many viewing it as critical for individuals lacking other treatment options, the decision has drawn attention to the role of patient advocacy in drug approvals for rare ailments.
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