BioBrief — Friday, April 24. Today’s episode covers four major biopharma developments: Regeneron’s FDA approval of the first gene therapy for genetic hearing loss, Sanofi’s positive CHMP opinion for progressive MS, Compass Pathways’ faster FDA review path for psilocybin, and Arrowhead’s positive European opinion for a rare-disease siRNA therapy.

1. Regeneron’s Otarmini becomes the first approved gene therapy for severe genetic hearing loss caused by OTOF mutations. In the CHORD trial, 16 of 20 efficacy patients met the main hearing-improvement endpoint at 24 weeks, with five of 12 reaching normal hearing on longer follow-up. The main significance is platform validation for inner ear gene therapy.
2. Sanofi’s tolebrutinib received a positive CHMP opinion for non-relapsing secondary progressive MS. In  ...