Otarmeni Regeneron’s first genetic medicine to gain approval

The US Food and Drug Administration (FDA) has approved Regeneron’s Otarmeni (lunsotogene parvec-cwha) as the first dual adeno-associated virus (AAV) vector-based gene therapy.

The in vivo gene therapy is authorised for children and adults with severe-to-profound and profound OTOF gene-associated sensorineural hearing loss, an ultra-rare condition. Specifically, it is available for patients with preserved outer hair cell function and no prior cochlear implant in the same ear.

The FDA’s decision represents the fastest Biologics License Application (BLA) approval in its modern history. It was granted based on data showing hearing sensitivity improvement by average pure tone audiometry at week 24 in the ongoing phase I/II CHORD trial. A total of 24 paediatric patients aged 10 months to 16 years were given Otarmeni via intracochlear infusion.

Of the 20 evaluable patients (efficacy), 80 percent gained improved hearing. FDA noted that this is “not expected in the natural history of the disease without intervention”. Additionally, longer follow-up demonstrated that 42 percent participants achieved normal hearing.

Continued approval for this indication may be subject to observed clinical benefit in the confirmatory portion of the CHORD clinical trial.

The FDA approval of Otarmeni signals a new era in the treatment of genetic forms of hearing loss, where reinstating 24/7 natural hearing is now possible”

Dr Aiden Eliot Shearer, Associate Professor of Otolaryngology-Head and Neck Surgery at Harvard Medical School and a CHORD trial investigator said: “The FDA approval of Otarmeni signals a new era in the treatment of genetic forms of hearing loss, where reinstating 24/7 natural hearing is now possible.

“In the pivotal trial, the one-time gene therapy demonstrated rapid, meaningful and consistent hearing responses, with most children achieving remarkable hearing improvements.”

Prior to this approval of Otarmeni, Regeneron advanced its ambitions in in vivo gene therapy in late 2025, through a partnership agreement with Tessera Therapeutics. The collaboration involves developing an investigational treatment for alpha-1 antitrypsin deficiency (AATD).