FDA Approves First Gene Therapy for Genetic Hearing Loss

The Food and Drug Administration approved lunsotogene parvec-cwha (Otarmeni, Regeneron Pharmaceuticals) April 23 to treat pediatric and adult patients with severe-to-profound sensorineural hearing loss associated with biallelic variants in the OTOF gene. 

OTOF-related hearing loss is caused by mutations that prevent production of otoferlin, a protein essential for synaptic transmission of sound signals in inner hair cells.  

Lunsotogene parvec-cwha is the first dual adeno-associated virus serotype 1 (AAV1) vector gene therapy approved by FDA and is administered as a one-time surgical dose into the cochlea. It delivers a functional copy of the OTOF gene to restore otoferlin expression and auditory signaling. It is the first disease-modifying therapy for this condition and is indicated for patients with preserved outer hair cell function and no prior cochlear implant in the treated ear. 

FDA based its approval on results from an ongoing, single-arm clinical trial in pediatric patients with OTOF-associated hearing loss. Among 20 evaluable patients, 80% experienced improved hearing after treatment. 

The application was granted Orphan Drug, Fast Track, Rare Pediatric Disease and Regenerative Medicine Advanced Therapy designations. Continued approval may be contingent on confirmatory data to verify clinical benefit. 

FDA also noted that the approval was granted 61 days after biologics license application submission under the agency’s Commissioner’s National Priority Voucher Pilot Program. It is the first gene therapy approved under the program and among the fastest BLA approvals on record. The agency will host a public meeting on June 4 to solicit additional feedback about the CNPV program.