FDA Grants Accelerated Approval to First Gene Therapy for Genetic Hearing Loss
Published: 07 May 2026
Author: Towards Healthcare
Regeneron Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Otarmeni, the first gene therapy and second new molecular entity approved under the FDA Commissioner’s National Priority Voucher program.
It is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss associated with molecularly confirmed biallelic variants in the OTOF gene, preserved outer hair cell function, and no prior cochlear implant in the same ear.
According to Towards Healthcare, the hearing loss gene therapy market is projected to experience significant growth, with estimates suggesting the market size will increase from USD 1.33 billion in 2026 to approximately USD 6.32 billion by 2035, representing a compound annual growth rate (CAGR) of 18.9% from 2026 to 2035. Growth is driven by the increasing number of individuals with genetic hearing impairments is a significant driver of market growth. Innovations in gene-editing technologies and delivery platforms are enhancing the accuracy and safety of gene therapy, making it a more viable option for treating hearing loss.
About Otarmeni (lunsotogene parvec-cwha)
Otarmeni is an adeno-associated virus vector-based gene therapy indicated for the treatment of certain pediatric and adult patients with severe-to-profound sensorineural hearing loss caused by variants of the OTOF gene that produce non-functional otoferlin protein.
These variants prevent production of otoferlin, a protein essential for transmitting sound signals from inner ear hair cells to the auditory nerve, resulting in congenital deafness despite intact cochlear structures.
About the CHORD trial
The CHORD trial is an ongoing, registrational Phase 1/2 multicenter, open-label trial to evaluate the safety, tolerability, and efficacy of Otarmeni in infants, children, and adolescents with OTOF-related hearing loss. The therapy delivers a functional OTOF gene via adeno-associated virus (AAV) vectors, aiming to restore natural acoustic hearing.
The trial is currently enrolling children across various sites in the U.S., the United Kingdom, and Spain.
A recent report by Towards Healthcare highlights that the hearing loss gene therapy market is witnessing growth driven by the increasing prevalence of genetic hearing impairment and the limited success of existing treatment options. Gene therapy offers a potential solution by correcting the underlying genetic defects in the inner ear, providing a more permanent and effective treatment than traditional devices like hearing aids or cochlear implants. These companies are investing in research and development, strategic partnerships, and commercialisation of landmark therapies, influencing pricing models and the pace of innovation across the industry.