RegulatoryApr 23, 2026, 12:42 PM
Regeneron's Otarmeni Gene Therapy Gets FDA Approval for Hearing Loss
AI Summary
Regeneron Pharmaceuticals announced that the FDA has granted accelerated approval for Otarmeni, its first gene therapy. This groundbreaking treatment is indicated for pediatric and adult patients with severe-to-profound OTOF-related sensorineural hearing loss. The approval is based on pivotal CHORD trial results, where 80% of participants met the primary endpoint and 42% achieved normal hearing, marking a significant advancement in treating genetic hearing loss.
Key Highlights
- FDA granted accelerated approval for Otarmeni (lunsotogene parvec-cwha).
- First gene therapy to restore neurosensory function to normal levels.
- CHORD trial: 80% achieved primary endpoint, 42% achieved normal hearing.
- Indicated for severe-to-profound OTOF-related hearing loss in pediatric and adult patients.
- Regeneron will make Otarmeni available for free in the U.S.