On April 23, 2026, the US Food and Drug Administration approved gene therapy Otarmeni (lunsotogene parvec-cwha) for the treatment of genetic hearing loss. Otarmeni, the first-ever gene therapy approved for this use, 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 otorfelin (OTOF) gene. About 50% of congenital hearing loss is caused by genetic mutations, and of those, 2% to 8% have variants in the OTOF gene. If patients have two non-working copies, they do not produce otoferlin, which disrupts sound signal transmission. OTOF hearing loss affects about 50 newborns per year in the United States.
Otarmeni is a one-time administration gene therapy that works by providing a copy of the OTOF gene to the inner hair cells to bring back otoferlin production and auditory signaling. It is given as a single dose in both cochleae via a syringe and catheter.
In the CHORD trial, Otarmeni was given to 20 participants. 80% noted hearing improvements per pure tone and audiometry assessments, and 70% had an auditory brainstem response. After 48 weeks, those who had responded had also sustained a response to therapy with 42% having achieved normal hearing.
Regeneron Pharmaceuticals, Inc., will provide Otarmeni free of charge for patients in the US. This will not include administration costs associated with receiving the gene therapy.
Article written by Stacy Schumacher, RN, BSN, CMCN, Managed Care Specialist. The following sources were used as reference material for this article: