The FDA has granted accelerated approval to Regeneron’s Otarmeni (lunsotogene parvec-cwha), positioning it as the first gene therapy designed to restore neurosensory function to normal levels in OTOF-related hearing loss. The therapy is indicated for children and adults with severe-to-profound and profound sensorineural hearing loss tied to biallelic OTOF variants, with preserved outer hair cell function and no prior cochlear implant in the same ear. Approval was based on hearing sensitivity improvements measured by average pure tone audiometry (PTA) at week 24 in the Phase I/II CHORD trial (NCT05788536). In trial results, 80% (16 of 20) met the primary endpoint at ≤70 dB HL at week 24, and 70% (14 of 20) met a key secondary endpoint on auditory brainstem response (ABR) at ≤90 dB. Regeneron said it will make Otarmeni available for free in the U.S., and noted the accelerated approval pathway may require verification and description of clinical benefit in a confirmatory portion of CHORD. The approval also came under the FDA’s Commissioner’s National Priority Voucher (CNPV) pilot program, reflecting a targeted regulatory push for high-need therapies.