Regeneron wins first FDA approval for genetic hearing loss gene therapy

The U.S. FDA has granted accelerated approval to Regeneron’s Otarmeni, marking the first gene therapy and second new molecular entity approved under the FDA Commissioner’s National Priority Voucher program.

Otarmeni 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. The treatment aims to deliver a working copy of the OTOF gene to replace the non-functional otoferlin protein using a modified, non-pathogenic virus that is delivered via an infusion into the cochlea under general anesthesia (similar to the procedure used for cochlear implantation).

OTOF-related hearing loss is an ultra-rare condition, affecting about 50 newborns per year in the U.S. Otarmeni is the first and only in vivo gene therapy for OTOF-related hearing loss and will be made available by Regeneron for free in the U.S.

The FDA approval is based on results from the pivotal CHORD trial, in which 20 participants (aged 10 months to 16 years) received a single dose of Otarmeni via intracochlear infusion, either unilaterally or bilaterally. In the trial, 80% of participants (16 of 20) experienced hearing improvements per pure tone audiometry assessments at a threshold of ≤70 dB HL at 24 weeks, achieving the trial’s primary endpoint; one additional participant achieved this threshold by week 48.