Regeneron Unveils 'First-and-Only' Otarmeni™ (lunsotogene parvec-cwha) for the Treatment of Genetic Hearing Loss
Regeneron Pharmaceuticals has introduced Otarmeni™ (lunsotogene parvec-cwha), the first and only treatment for OTOF-related genetic hearing loss in pediatric and adult patients with severe-to-profound sensorineural hearing loss.
Otarmeni is an adeno-associated virus (AAV) vector-based gene therapy designed to deliver a functional copy of the OTOF gene directly to cochlear hair cells, with the aim of restoring physiological hearing. The therapy is indicated for patients with molecularly confirmed biallelic OTOF gene variants, preserved outer hair cell function and no prior cochlear implant in the treated ear.
The FDA approval was granted under the accelerated approval pathway based on results from the pivotal Phase 1/2 CHORD clinical trial. The study demonstrated clinically meaningful improvements in hearing sensitivity, with 80% of participants achieving significant hearing improvement at 24 weeks and 70% demonstrating objective hearing function based on auditory brainstem response assessments. Among participants followed for 48 weeks, treatment responses were maintained, with some achieving hearing levels within the normal range.
OTOF-related hearing loss is an ultra-rare genetic disorder caused by mutations in the OTOF gene, resulting in the absence of functional otoferlin protein required for communication between inner ear sensory cells and the auditory nerve. The condition has traditionally been managed with hearing devices such as cochlear implants, without addressing the underlying genetic cause.
Otarmeni has received Orphan Drug, Rare Pediatric Disease, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations from the U.S. FDA. The therapy is also under regulatory review for additional global markets while the ongoing CHORD trial continues to evaluate its long-term safety and clinical benefit.
