First-in-class Otarmeni gene therapy could restore hearing in rare OTOF-related loss, Regeneron to provide free US access

Regeneron’s Otarmeni (lunsotogene parvec-cwha) received FDA accelerated approval as the first in vivo gene therapy for OTOF-related severe-to-profound hearing loss, based on CHORD trial results showing 80% reached a PTA threshold of ≤70 dB HL at 24 weeks and 42% achieved normal hearing (≤25 dB HL) by 48 weeks. The one-time intracochlear infusion is intended for patients with biallelic OTOF variants, preserved outer hair cell function, and no prior cochlear implant in the treated ear, and Regeneron will provide Otarmeni at no cost to eligible U.S. patients. Safety signals include otitis media, dizziness and other procedure-related events; continued approval will depend on confirmatory CHORD data and site-specific eligibility constraints.