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On April 28, 2026, the U.S. Food and Drug Administration approved Otarmeni (lunsotogene parvec-cwha), the world’s first dual adeno-associated virus (AAV) vector-based gene therapy for treating severe-to-profound sensorineural hearing loss associated with biallelic OTOF gene variants. The therapy was developed by Regeneron Pharmaceuticals.
The approval marks several milestones: it was granted just 61 days after the Biologics License Application (BLA) was filed, tying for the fastest BLA approval in modern FDA history. It is also the sixth product approved under the Commissioner’s National Priority Voucher (CNPV) pilot program and the first gene therapy product to receive approval under the program.
Genetic mutations cause approximately half of all congenital hearing loss. OTOF gene variants account for 2% to 8% of inherited, non-syndromic cases. Patients with two non-functional copies of the gene cannot produce otoferlin, a protein essential for sound signal transmission. Prior to this approval, no disease-modifying treatments existed for OTOF-related deafness.
Otarmeni is a one-time biologic-device combination product administered surgically as a single dose per ear into the cochlea. It delivers a functional copy of the OTOF gene to inner hair cells, restoring otoferlin production and auditory signaling.
In a clinical trial involving 24 patients aged 10 months to 16 years, 80% of the 20 evaluable patients (16 individuals) experienced improved hearing — an outcome not expected in the natural history of the disease without intervention.
FDA Commissioner Marty Makary, M.D., M.P.H., said: “Today’s approval is a significant milestone in the treatment of genetic hearing loss. Through the national priority voucher pilot program, we are accelerating therapies for rare diseases with unmet medical needs while proving we can successfully review even the most complex submissions — such as novel dual vector gene therapies and combination products requiring coordination across multiple offices and centers — in significantly shortened timeframes.”
Common side effects included middle ear infection, nausea, dizziness, and procedural pain. The therapy has received orphan drug, rare pediatric disease, fast track, and regenerative medicine advanced therapy (RMAT) designations.
Regeneron will continue to collect data on the durability of hearing improvement and verification of treatment effects on clinical measures of speech development and quality of life ahead of the FDA’s public meeting on the CNPV program scheduled for June 4.
Source: FDA, New England Journal of Medicine