On 23 April 2026, the United States Food and Drug Administration approved Otarmeni (lunsotogene parvec-cwha), the first dual adeno-associated virus vector-based gene therapy for inherited hearing loss. The therapy is developed for severe-to-profound sensorineural hearing loss linked to biallelic variants in the OTOF gene.
What Is OTOF-Related Hearing Loss?
OTOF-related hearing loss is a genetic disorder caused by mutations in the OTOF gene, which encodes otoferlin, a protein needed for auditory signalling in inner hair cells. The condition is classified as an ultra-rare form of inherited deafness and affects about 50 newborns each year in the United States.
How The Therapy Works
Otarmeni delivers a functional copy of the OTOF gene to inner hair cells through dual AAV vectors. The treatment restores otoferlin protein production and supports transmission of sound signals in the cochlea. It is administered as a single dose per ear by surgical delivery into the cochlea.
Clinical Trial And Eligibility
- The pivotal CHORD trial recorded improved hearing in 80% of evaluable participants.
- Normal hearing, including detection of whispers, was achieved by 42% of evaluable participants.
- Sixteen of 20 children showed hearing improvement about five months after treatment.
- The approved use covers paediatric and adult patients with preserved outer hair cell function and no prior cochlear implant in the treated ear.
Regulatory And Programme Details
This approval is the sixth under the FDA Commissioner’s National Priority Voucher pilot programme. It was granted 61 days after filing and is tied for the fastest Biologics License Application approval in modern FDA history.
Related Medical Context
Before this approval, management options for this condition included hearing aids and cochlear implants. Regeneron Pharmaceuticals has stated that Otarmeni will be offered at no cost to eligible patients in the United States.
Last Modified: April 24, 2026