FDA Grants Accelerated Approval to Otarmeni, First Gene Therapy for Genetic Hearing Loss

The U.S. Food and Drug Administration has granted accelerated approval to Otarmeni (lunsotogene parvec-cwha), the first-ever gene therapy approved for the treatment of genetic hearing loss. This approval marks a significant milestone as the first disease-modifying treatment available for patients with hearing loss caused by biallelic variants in the OTOF gene.

Otarmeni is indicated for 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. The therapy is intended for patients with preserved outer hair cell function and no prior cochlear implant in the same ear.

The approval follows the publication of results from the CHORD trial in the New England Journal of Medicine, which demonstrated that 80% of participants achieved or surpassed the primary hearing endpoint. With longer follow-up, 42% of participants achieved normal hearing levels, including the ability to hear whispers.

Prior to this approval, no disease-modifying treatments existed for OTOF-related deafness. Genetic mutations in the OTOF gene account for 2% to 8% of inherited, non-syndromic cases of congenital hearing loss. Patients with two nonworking copies of the OTOF gene do not produce otoferlin, a protein essential for transmitting sound signals from the inner ear to the brain.

Otarmeni is a dual adeno-associated virus (AAV) vector-based gene therapy designed to deliver functional copies of the OTOF gene to the inner ear. The FDA’s approval was granted under its National Priority Voucher (CNPV) pilot program, which aims to accelerate therapies for rare diseases with unmet medical needs. This marks the sixth approval under the CNPV program and the first gene therapy product approved via this pathway.

The FDA reviewed Otarmeni’s biologics license application in 61 days, tying it for the fastest BLA approval in modern FDA history. The agency noted that the approval reflects its ability to review complex submissions, such as novel dual-vector gene therapies, within significantly shortened timeframes when addressing critical public health needs.

FDA Commissioner Marty Makary, M.D., M.P.H., stated that the approval represents a significant advancement in treating genetic hearing loss and underscores the agency’s commitment to accelerating access to innovative therapies for rare diseases through regulatory pathways like the national priority voucher program.