FDA Approves Otarmeni, the First Gene Therapy for Genetic Hearing Loss
The U.S. FDA has approved Otarmeni (lunsotogene parvec-cwha), Regeneron Pharmaceuticals’ gene therapy for severe-to-profound sensorineural hearing loss caused by biallelic OTOF gene variants.
Otarmeni is the first approved gene therapy for genetic hearing loss and the first dual-AAV vector-based gene therapy to reach approval. The decision is notable not only for rare disease innovation, but also for showing how gene therapy is moving into the restoration of sensory function.
Key points
- First approved gene therapy for OTOF-related hearing loss
- Approved for pediatric and adult patients
- One-time dual-AAV gene therapy delivered to the cochlea
- Approved under the accelerated approval pathway
- First gene therapy approved under the FDA Commissioner’s National Priority Voucher program
In the CHORD trial, 80% of participants met the primary endpoint at 24 weeks, and longer follow-up showed that some patients improved to hearing levels associated with whisper perception.
We extend our congratulations to Regeneron Pharmaceuticals and to everyone involved in bringing this therapy forward.