At a Glance
- Company: Regeneron
- Location: US
- Approval: FDA accelerated, 23 April 2026
- Indication: OTOF biallelic mutation hearing loss
- Next steps: Free US supply, confirmatory trials
What happened?
The FDA approved Otarmeni™ (lunsotogene parvec-cwha), Regeneron’s AAV gene therapy, on 23 April 2026 as the first treatment for genetic hearing loss. Granted accelerated approval via the Commissioner’s National Priority Voucher, it targets severe-to-profound bilateral congenital or childhood-onset sensorineural hearing loss from OTOF mutations in patients without prior cochlear implants.
Why does it matter?
Otarmeni delivers functional otoferlin gene to inner ear hair cells via single bilateral cochlear injection, restoring natural hearing without devices. In rare OTOF deafness (~5% genetic causes, ~50 US births/year), CHORD trial showed 80% meaningful improvement; 42% normal hearing thresholds.
Who’s behind it?
Regeneron (post-2023 Decibel Therapeutics acquisition) developed the AAV1-Anc80.L651 vector. Boston Children’s A. Eliot Shearer led CHORD; Regeneron will provide free US treatment and partner for global access.
How was it achieved?
Phase 1/2 CHORD (n=12) met primary endpoint: 11/12 patients with ≥9 dB 4-frequency average improvement at 24 weeks. Sustained gains through 52 weeks; safety profile consistent with AAV cochlear delivery.
What’s next?
Confirmatory CHORD extension; EMA PRIME/Orphan status. Regeneron funds free US access; platform eyes expanded genetic hearing losses.
What are people saying?
A. Eliot Shearer, MD, PhD: “New era… children hearing voices, music naturally.” Regeneron: “First gene therapy restoring neurosensory function to normal.”