Accelerated Approval Granted to Gene Therapy for Rare Genetic Hearing Loss by Madaline Spencer| Published on: Apr 29, 2026

Accelerated Approval Granted to Gene Therapy for Rare Genetic Hearing Loss by Madaline Spencer| Published on: Apr 29, 2026 The US Food and Drug Administration (FDA) has granted accelerated approval for Otarmeni (lunsotogene parvec) for the treatment of patients with otoferlin (OTOF)-related hearing loss. The indication includes pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. https://checkrare.com/accelerated-approval-granted-to-gene-therapy-for-rare-genetic-hearing-loss/