Gene Therapy Briefs: Regeneron Wins FDA Approval for First Neurosensory Gene Therapy

The U.S. Food and Drug Administration (FDA) approved Regeneron Pharmaceuticals’ Otarmeni™ (lunsotogene parvec-cwha) as the first gene therapy designed to restore hearing in patients with severe-to-profound sensorineural hearing loss caused by biallelic variants in the *OTOF* gene. The therapy targets children and adults with preserved outer hair cell function and no prior cochlear implant in the treated ear, offering a potential alternative to cochlear implants. Approval was based on Phase I/II CHORD trial data, where 20 participants aged 10 months to 16 years received a single intracochlear infusion of Otarmeni. By week 24, 80% (16 of 20) showed improved hearing sensitivity to ≤70 dB HL, and 70% (14 of 20) demonstrated an auditory brainstem response at ≤90 dB. Among those followed to 48 weeks, 42% (five of 12) achieved normal hearing, including whispers at ≤25 dB HL. Regeneron stated Otarmeni will be provided for free in the U.S. The FDA’s accelerated approval requires confirmation of clinical benefit in the ongoing CHOIRD trial, a first-in-human study assessing safety, tolerability, and efficacy in infants, children, and adolescents with *OTOF*-related hearing loss. This approval marks the first gene therapy and second new molecular entity under the FDA’s Commissioner’s National Priority Voucher (CNPV) program, launched in October 2023 to expedite reviews for therapies addressing critical health needs. The CNPV program, led by FDA Commissioner Martin A. Makary, MD, allows faster application reviews (1–2 months instead of 10–12) for drugs meeting unmet medical needs, including domestic manufacturing for national security. Regeneron’s Otarmeni represents a landmark achievement in gene therapy for neurosensory disorders, offering hope for patients previously limited to cochlear implants or hearing aids.