The FDA granted accelerated approval to Regeneron’s in vivo gene therapy Otarmeni (lunsotogene parvec-cwha), marking the first gene therapy intended to restore neurosensory function to normal levels for severe-to-profound sensorineural hearing loss in patients with biallelic OTOF variants. Regeneron previously developed Otarmeni as DB-OTO and tested it in the CHORD Phase I/II trial (NCT05788536). Approval was based on hearing sensitivity improvements at week 24 measured by average pure tone audiometry (PTA). Regeneron reported 80% of participants (16 of 20) met the primary endpoint at ≤70 dB HL by week 24, with 70% (14 of 20) achieving an auditory brainstem response (ABR) at ≤90 dB decibels. Among responders followed to 48 weeks, Regeneron said response was maintained. Regulators tied accelerated approval to confirmatory verification of clinical benefit in a planned part of CHORD. Regeneron also said it will make Otarmeni available for free in the U.S., and the approval is under the FDA Commissioner’s National Priority Voucher (CNPV) pilot program. For the field, Otarmeni’s approval expands the practical footprint of AAV-based gene therapy into pediatric neurosensory indications, strengthening investor and developer focus on durable functional restoration when a clear functional endpoint is feasible for review.