Otarmeni™ (lunsotogene parvec-cwha) Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss; Regeneron to Provide Otarmeni for Free in the U.S.
Regeneron • • business
Key Points:
- The FDA has granted accelerated approval to Otarmeni™ (lunsotogene parvec-cwha), developed by Regeneron, as the first gene therapy to restore natural hearing in patients with severe-to-profound sensorineural hearing loss caused by biallelic OTOF gene variants.
- The approval is based on the CHORD trial results, where 80% of participants met the primary endpoint of improved hearing sensitivity at 24 weeks, and 42% achieved normal hearing including whispers with longer follow-up.
- Otarmeni is delivered via a single intracochlear infusion and is indicated for pediatric and adult patients with no prior cochlear implant in the treated ear, preserving outer hair cell function; Regeneron will provide the therapy at no cost to eligible U.S. patients.
- The gene therapy represents a significant advancement in treating genetic hearing loss, historically managed only with hearing devices, and offers a potential durable restoration of physiological hearing by delivering a functional OTOF gene.
- Common side effects include middle ear infections, nausea, dizziness, and procedural pain, and the therapy requires administration by surgeons trained in intracochlear surgery; continued approval depends on confirmatory trial results.
