First Gene Therapy Approved
- The FDA approved the first gene therapy for inherited hearing loss, Regeneron's Otarmeni (lunsotogene parvec-cwha). - Regeneron said it will provide the one-time treatment for free in the United States. - Trial results reported 80% meeting or surpassing the primary hearing endpoint, with 42% achieving normal hearing including whispers (cnn.com).
Gene therapy works by delivering a working copy of a faulty gene into the cells that need it. On April 23, the Food and Drug Administration approved Otarmeni for a rare inherited form of deafness tied to the OTOF gene. (fda.gov) The treatment is for children and adults with severe-to-profound or profound sensorineural hearing loss caused by confirmed mutations in both copies of OTOF. The Food and Drug Administration said it is the first-ever dual adeno-associated virus gene therapy and the first approved for this kind of genetic hearing loss. (fda.gov) OTOF helps inner-ear cells make otoferlin, a protein that lets sound signals pass from the ear to the hearing nerve. When that protein is missing, the ear’s structure can remain in place but the signal does not get through. (nejm.org) Otarmeni is given once, by surgery into the cochlea, the spiral structure in the inner ear that turns vibrations into nerve signals. The approved label says each ear receives a single intracochlear infusion of 7.2 × 10^12 vector genomes in 0.24 milliliters. (regeneron.com) The approval was based on the CHORD trial, a Phase 1/2 study that began in June 2023 and is still recruiting, with an estimated enrollment of 30 participants. Regeneron said 80% of treated participants reached or beat the main hearing target, and 42% later achieved hearing in the normal range, including whispers. (clinicaltrials.gov) (regeneron.com) A March 12, 2026 paper in The New England Journal of Medicine reported that DB-OTO, the earlier name for Otarmeni, improved hearing in 12 treated patients and normalized hearing sensitivity in 3 of them. The Food and Drug Administration said no disease-modifying treatment had existed for OTOF-related deafness before this approval. (nejm.org) (fda.gov) The agency granted accelerated approval, which means the treatment reached the market based on an earlier measure of benefit rather than completed confirmatory evidence. Regeneron’s label says continued approval may depend on the rest of the CHORD trial confirming clinical benefit. (fda.gov) (regeneron.com) The Food and Drug Administration said the review moved unusually fast under its Commissioner’s National Priority Voucher pilot, with approval coming 61 days after the biologics license application filing. The agency called it the first gene therapy cleared under that program and tied for the fastest biologics license approval in modern Food and Drug Administration history. (fda.gov) Regeneron said it will provide Otarmeni free in the United States. The company also said the treatment is its first approved genetic medicine. (regeneron.com) The label also sets limits on who can get it: patients need preserved outer hair cell function, and the same ear cannot already have a cochlear implant. Common side effects listed in the prescribing information include middle-ear infection, vomiting, nausea, dizziness, procedural pain, gait disturbance, and nystagmus. (regeneron.com)
