The FDA granted accelerated approval to Regeneron’s in vivo gene therapy Otarmeni (lunsotogene parvec-cwha), positioning it as the first gene therapy intended to restore neurosensory hearing in OTOF-related hearing loss. Otarmeni is indicated for children and adults with severe-to-profound or profound sensorineural hearing loss tied to molecularly confirmed biallelic OTOF variants, with eligibility requirements including preserved outer hair cell function and no prior cochlear implant in the treated ear. The agency based its decision on hearing-sensitivity improvement measured by average pure tone audiometry (PTA) at week 24 in the Phase I/II CHORD trial (NCT05788536). In the trial’s 20 participants (10 patients treated in one ear and 10 in both ears), 80% reported hearing improvements at week 24 at ≤70 dB HL, and 70% achieved an auditory brainstem response (ABR) threshold at ≤90 decibels. Regeneron said confirmatory evidence will be required in the follow-on portion of the CHORD/CHOIRD program to verify and describe clinical benefit. Otarmeni also received priority access under the agency’s Commissioner’s National Priority Voucher (CNPV) pilot program, and Regeneron plans to make the therapy available free in the U.S.