FDA approves rare disease drug Forzinity despite reviewer concerns about effectiveness

The FDA approved a costly rare disease drug despite internal reviewer objections regarding its effectiveness compared to a placebo. The drug, Forzinity, for Barth syndrome, priced at nearly $800,000 annually, received accelerated approval based on urgent patient need and some observed motor skill improvements. Nearly a dozen reviewers recommended against approval, citing insufficient evidence of effectiveness, but an FDA office chief ultimately authorized it, citing a study showing improved knee strength.