FDA approves Otarmeni, first gene therapy for genetic hearing loss caused by OTOF gene mutations, in record 61 days; 80% of pediatric patients showed improved hearing in trial

FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss Under National Priority Voucher Program

FDA News Release Groundbreaking AAV-based gene therapy offers potential treatment for patients with OTOF gene-associated severe-to-profound and profound hearing loss For Immediate Release: April 23, 2026 The U.S. Food and Drug Administration today 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 ...