FDA Approves Controversial Rare Disease Drug Despite Debates

The FDA approved a $800,000 rare disease drug, Forzinity, for Barth syndrome despite findings questioning its efficacy. The decision was supported by urgent patient need and slight improvements in studies, though some reviewers opposed it. Patients and advocates pressured approval due to limited treatment options.

In a controversial move, the U.S. FDA has given the green light for Forzinity, a costly drug aimed at treating Barth syndrome, despite data reviewers concluding its effects weren't markedly superior to a placebo. The decision on the drug, priced at nearly $800,000 annually, came after much debate.

FDA's documents reveal that the approval of Stealth Biotherapeutics' elamipretide—marketed as Forzinity—was driven by an urgent unmet need in patients, albeit with reservations about its efficacy. The disease, affecting around 150 U.S. males, often results in severe infancy heart failure.

The approval followed advocacy pressures from lawmakers and patient groups, with Forzinity marking the first treatment of its kind for Barth syndrome. However, internal disagreements within the FDA highlight ongoing tensions over balancing scientific evidence against patient demands for ultra-rare disease solutions.

(With inputs from agencies.)