Regeneron Pharmaceuticals Inc. (REGN), a biotechnology company, on Friday reported the acceptance of a Market Authorization Application (MAA) review by the European Medicines Agency (EMA) for Otarmeni, a gene therapy developed to treat OTOF-associated hearing loss.
Biallelic OTOF variant-associated hearing loss is caused by mutations in the OTOF gene, leading to the absence of a functioning otorfelin protein. The protein is essential in communications between the auditory nerve and sensory cells of the inner year. Treated by the use of hearing devices that can amplify sound but fails to restore hearing across the spectrum, OTOF-associated hearing loss was believed to be permanent.
Otarmeni (lunsotogene parvec) is an in vivo adeno-associated virus (AAV) vector-based gene therapy that delivers the functional OTOF gene to cells and facilitates otorfelin production.
The therapy received an accelerated approval from the U.S. Food and Drug Administration (FDA) in April 2026 and is the first gene therapy for the treatment of OTOF-associated hearing loss. The EMA also granted Otarmeni an orphan drug designation.
The MAA is supported by results from the ongoing registrational, multi-center, open-label Phase 1/2 CHORD trial, which is presently enrolling adults and children with OTOF-associated hearing loss.
The study consists of a dose-escalation cohort (Part A), wherein participants receive a single intracochlear infusion in one ear, and an expansion cohort (Part B), where patients receive the Part A selected dose in both ears. Improvement in hearing after the treatment is assessed by measuring average pure tone audiometry (PTA) and auditory brainstem response (ABR).
REGN is currently trading at $637.10, down 0.85%.
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