Regeneron won accelerated approval from the FDA for Otarmeni (lunsotogene parvec-cwha), described as the first gene therapy to restore neurosensory function for OTOF-associated sensorineural hearing loss. The approval covers children and adults with severe-to-profound or profound hearing loss with molecularly confirmed biallelic OTOF variants. The agency based its decision on hearing-sensitivity improvements at week 24 from the Phase I/II CHORD trial, including 80% of participants meeting the primary endpoint of ≤70 dB HL on pure tone audiometry. Regeneron said 42% of participants achieved normal hearing including whispers at 48 weeks among those followed. Regeneron positioned the approval as a significant expansion of in vivo AAV gene-therapy applications and said it will make Otarmeni available for free in the US, citing the FDA’s Commissioner’s National Priority Voucher program.