BioBrief — Thursday, April 23 Today’s episode covers three major biopharma developments: Regeneron’s FDA-approved gene therapy for inherited hearing loss, Novo Nordisk’s positive pediatric semaglutide data, and Grace Therapeutics’ FDA setback on its stroke drug. The common thread is that each story shifts regulatory probability, but in different directions: one clears a major hurdle, one expands an existing franchise, and one runs into manufacturing and toxicology friction.

1) Regeneron gene therapy approval The FDA approved Regeneron’s gene therapy for inherited hearing loss, marking the first approval in this category. The therapy, Otarmeni, is a dual AAV gene therapy that replaces the defective OTOF gene, which is needed for sound signal transmission in the inner ear. The approval was based on a small si ...