The FDA granted accelerated approval to Regeneron’s in vivo gene therapy Otarmeni (lunsotogene parvec-cwha), making it the first gene therapy designed to restore neurosensory function in OTOF-related sensorineural hearing loss. The approval covers children and adults with severe-to-profound hearing loss tied to molecularly confirmed biallelic OTOF variants, preserved outer hair cell function, and no prior cochlear implant in the treated ear. Otarmeni’s accelerated approval was based on results from the Phase I/II CHORD trial (NCT05788536). At week 24, 80% of participants (16 of 20) achieved hearing improvements to ≤70 dB HL on average pure-tone audiometry, meeting the primary endpoint, with 70% (14 of 20) reaching ≤90 dB HL on auditory brainstem response as a key secondary endpoint. Regeneron said that responses were maintained through follow-up at 48 weeks for those who were initial responders and reported that 42% of all participants (5 of 12 followed at 48 weeks) achieved “normal hearing” including whispers (≤25 dB HL). The company also said it will make Otarmeni available for free in the U.S., while confirming that confirmatory data are expected to further validate clinical benefit.