Otarmeni™ (lunsotogene parvec) Receives EMA Filing Acceptance for Genetic Hearing Loss

About this update from Regeneron Pharmaceuticals, Inc.

TARRYTOWN, N.Y., May 22, 2026 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the European Medicines Agency (EMA) has accepted for review under Accelerated Assessment the Marketing Authorization Application (MAA) for Otarmeni™ (lunsotogene parvec), an in vivo adeno-associated virus vector-based gene therapy for the treatment of biallelic OTOF variant-associated hearing loss. 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). The MAA is supported by data from the pivotal CHORD clinical trial, in which 24 participants (aged between 10 months to 16 years) received a single dose of Otarmeni via intracochlear infusion, either unilaterally (in one ear; n=10) or bilaterally (in both ears; n=14). An earlier cut of results from the CHORD trial (n=20) also supported the U.S. Food and Drug Administration’s recent accelerated approval of Otarmeni in April 2026. Regulatory submissions are planned in additional markets, including Japan. OTOF-related hearing loss is an ultra-rare condition, affecting approximately 46 newborn children per year in the EU. Though all structures within the ear are intact, variants in the OTOF gene cause a lack of a functional otoferlin protein, which is critical for communication between the sensory cells of the inner ear and the auditory nerve. Historically, genetic OTOF-related hearing loss was considered permanent and managed with life-long use of devices. While these devices can amplify sound to improve hearing for individuals with a range of hearing loss, they do not currently restore the full spectrum of sound. Otarmeni is approved in the U.S. Outside of the U.S., the safety and efficacy of Otarmeni have not been fully evaluated. About the CHORD TrialThe CHORD trial is an ongoing, registrational Phase 1/2 multicenter, open-label trial to evaluate the safety, tolerability and efficacy of Otarmeni in infants, children and adolescents with OTOF-related hearing loss. The trial is currently enrolling children and adults across sites in the U.S., United Kingdom, Spain, Germany and Japan. CHORD is being conducted in two parts. In the initial dose-escalation cohort (Part A), participants receive a single intracochlear infusion of Otarmeni in one ear. In the...

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