FDA Raises Concerns Over Intercept's NASH Treatment, Stock Drops.

The FDA has released briefing documents ahead of an advisory committee meeting stating that Intercept's Ocaliva cannot be justified for use in NASH subjects with Stage 2 or 3 fibrosis. This comes after the FDA rejected Intercept's push for accelerated approval in 2020. Meanwhile, X4 Pharmaceuticals has released more positive data from its Phase III study of mavorixafor as a treatment of WHIM syndrome, and PTC Therapeutics has claimed a Phase III win in rare genetic disease phenylketonuria. The FDA will also review Takeda's enzyme replacement therapy for an ultra-rare blood clotting disorder, and Canadian biotech enGene plans to combine with a Forbion-led blank check company to land a seat on Nasdaq and get to an FDA approval request in the first half of 2025.