The FDA granted accelerated approval to Regeneron’s Otarmeni (lunsotogene parvec-cwha), positioning it as the first gene therapy designed to restore neurosensory function for OTOF-related severe-to-profound sensorineural hearing loss. The in vivo AAV therapy is indicated for children and adults with molecularly confirmed biallelic OTOF variants and preserved outer hair cell function. Approval was based on hearing sensitivity improvements at 24 weeks in the Phase I/II CHORD trial (NCT05788536), with 80% (16 of 20) meeting the primary endpoint defined as ≤70 dB HL. Regeneron said a confirmatory portion of CHORD will verify and describe clinical benefit, as required under the FDA’s accelerated approval framework. Regeneron also said Otarmeni will be made available for free in the U.S., and the agency evaluated it under Commissioner’s National Priority Voucher (CNPV) program. The decision underscores continued regulatory openness to targeted gene therapies with clear functional endpoints.