The FDA granted accelerated approval to Regeneron’s Otarmeni (lunsotogene parvec-cwha), described as the first gene therapy intended to restore neurosensory function in OTOF-related sensorineural hearing loss. The in vivo AAV vector therapy is indicated for children and adults with severe-to-profound hearing loss tied to biallelic OTOF variants, with preserved outer hair cell function and no prior cochlear implant in the treated ear. The approval was based on week-24 improvements in hearing sensitivity measured by pure tone audiometry (PTA) in the Phase I/II CHORD trial (NCT05788536). Regeneron reported that 80% of participants (16 of 20) reached ≤70 dB HL at week 24, with additional week-48 responder confirmation. The FDA also cited an auditory brainstem response endpoint and noted ongoing needs for confirmatory evidence. Regeneron said Otarmeni will be made available for free in the U.S., and it is the first gene therapy (and second new molecular entity) to receive approval under the Commissioner’s National Priority Voucher (CNPV) pilot program. The confirmatory work is expected to be addressed in the trial’s ongoing structure. For the broader gene-therapy space, the approval reinforces regulators’ willingness to use accelerated pathways when functional endpoints show rapid, clinically meaningful signals in rare, genetically defined indications.