The FDA granted accelerated approval to Regeneron’s Otarmeni (lunsotogene parvec-cwha), positioning it as the first gene therapy designed to restore a neurosensory function to normal levels. Otarmeni is indicated for children and adults with severe-to-profound sensorineural hearing loss due to biallelic OTOF variants and no prior cochlear implant in the treated ear. The decision was based on hearing sensitivity improvements at week 24 from Regeneron’s Phase I/II CHORD trial (NCT05788536). In the study, 80% of participants (16 of 20) reached ≤70 dB HL on pure-tone audiometry at 24 weeks, and 70% (14 of 20) achieved ≤90 dB HL on auditory brainstem response. Regeneron said it will make Otarmeni available for free in the U.S. The agency tied the approval to confirmatory evidence from a CHOIRD trial, including a first-in-human validation framework under its Commissioner’s National Priority Voucher (CNPV) pilot program.