Groundbreaking AAV-based gene therapy offers potential treatment for patients with OTOF gene-associated severe-to-profound and profound hearing loss
The U.S. Food and Drug Administration on April 23, 2026, approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy. Otarmeni is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene.
Following the publication of powerful results of hearing restoration in the New England Journal of Medicine, the FDA acted swiftly to grant a national priority voucher for an accelerated review. Today’s approval was issued 61 days after BLA filing, marking the sixth approval under the Commissioner’s National Priority Voucher (CNPV) pilot program and the first gene therapy product approved under the program. It is also tied for the fastest BLA approval in modern FDA history.
