Regeneron Pharmaceuticals announced the European Medicines Agency (EMA) has accepted for review its marketing authorization application (MAA) for Otarmeni (lunsotogene parvec). The application for the gene therapy, which treats biallelic OTOF variant-associated hearing loss, is being reviewed under the EMA’s Accelerated Assessment pathway.

Otarmeni, formerly known as DB-OTO, previously received Orphan Designation from the EMA. If approved, Otarmeni will be the first gene therapy for OTOF-related hearing loss in the European Union (EU), according to the company.

New DB-OTO Results Show Sustained Improvements for Children with Profound Genetic Hearing Loss

Otarmeni is an in vivo adeno-associated virus vector-based gene therapy designed to address hearing loss caused by variants in the OTOF gene. This ultra-rare condition, affecting approximately 46 newborns annually in the European Union, results from the lack of a functional otoferlin protein, which is necessary for communication between inner ear sensory cells and the auditory nerve. The therapy delivers a working copy of the OTOF gene via an intracochlear infusion.

Supporting Clinical Data

The MAA is supported by data from the pivotal CHORD clinical trial. In the trial, 24 participants, aged 10 months to 16 years, received a single dose of Otarmeni. Data from the CHORD trial also supported the US Food and Drug Administration’s accelerated approval of Otarmeni in April 2026. Regeneron plans regulatory submissions in additional markets, including Japan.

FDA Grants Accelerated Approval to Otarmeni Gene Therapy for OTOF-Related Hearing Loss

About the CHORD Trial

The CHORD trial is an ongoing Phase 1/2 multicenter, open-label study evaluating the safety, tolerability, and efficacy of Otarmeni in infants, children, and adolescents. The trial consists of an initial dose-escalation cohort and an expansion cohort where participants receive the therapy in both ears. Hearing improvements were evaluated using pure tone audiometry (PTA) and auditory brainstem response (ABR). According to the company, all participants had profound hearing loss and no electrophysiological (ABR) responses at maximum sound levels at baseline.

For more information about Otarmeni gene therapy for OTOF-related hearing loss, visit https://www.regeneron.com.