{"id":954311,"date":"2026-04-23T12:43:06","date_gmt":"2026-04-23T16:43:06","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/otarmeni-lunsotogene-parvec-cwha-approved-by-fda-as-first-and-only-gene-therapy-for-genetic-hearing-loss-regeneron-to-provide-otarmeni-for-free-in-the-u-s\/"},"modified":"2026-04-23T12:43:06","modified_gmt":"2026-04-23T16:43:06","slug":"otarmeni-lunsotogene-parvec-cwha-approved-by-fda-as-first-and-only-gene-therapy-for-genetic-hearing-loss-regeneron-to-provide-otarmeni-for-free-in-the-u-s","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/otarmeni-lunsotogene-parvec-cwha-approved-by-fda-as-first-and-only-gene-therapy-for-genetic-hearing-loss-regeneron-to-provide-otarmeni-for-free-in-the-u-s\/","title":{"rendered":"Otarmeni\u2122 (lunsotogene parvec-cwha) Approved by FDA as First and Only Gene Therapy for Genetic Hearing Loss; Regeneron to Provide Otarmeni for Free in the U.S."},"content":{"rendered":"
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\n Approval in severe-to-profound and profound\u00a0<\/strong>
\n
\n OTOF<\/em>
\n <\/strong>
\n -related hearing loss is based on pivotal results of the CHORD trial demonstrating 80% of participants achieved or surpassed a hearing level meeting the primary endpoint, and with longer follow-up, 42% achieved normal hearing that included whispers<\/strong>\n <\/p>\n

\n First FDA-approved example of a gene therapy to restore a neurosensory function to normal levels<\/strong>\n <\/p>\n

\n Otarmeni is Regeneron\u2019s first approved genetic medicine, showcasing its ability to advance new therapeutic approaches to address conditions with great unmet medical need <\/strong>\n <\/p>\n

TARRYTOWN, N.Y., April 23, 2026 (GLOBE NEWSWIRE) — Regeneron Pharmaceuticals, Inc.\u00a0(NASDAQ: REGN) today announced the\u00a0U.S. Food and Drug Administration\u00a0(FDA) has granted accelerated approval for Otarmeni\u2122 (lunsotogene parvec-cwha), the first gene therapy and second new molecular entity approved under the FDA Commissioner\u2019s National Priority Voucher program. Otarmeni is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 decibel hearing level [dB HL]) associated with molecularly confirmed biallelic variants in the OTOF <\/em>gene, preserved outer hair cell function, and no prior cochlear implant in the same ear. Otarmeni, formerly known as DB-OTO, is the first and only in vivo<\/em> gene therapy for OTOF<\/em>-related hearing loss and will be made available by Regeneron for free in the U.S.<\/p>\n

Otarmeni was granted accelerated approval based on the improvement of hearing sensitivity by average pure tone audiometry (PTA) at week 24. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory portion of the CHORD clinical trial. Otarmeni is not recommended in patients in whom preoperative imaging demonstrates that access to the inner ear is not feasible, including those with abnormal mastoid pneumatization or clinically significant anatomic variations of the middle ear and inner ear. <\/p>\n

\u201cThe FDA approval of Otarmeni signals a new era in the treatment of genetic forms of hearing loss, where reinstating 24\/7 natural\u00a0hearing is now possible,\u201d said A. Eliot Shearer, M.D., Ph.D., otolaryngologist in the department of Otolaryngology and Communication Enhancement at Boston Children\u2019s Hospital, Associate Professor of Otolaryngology-Head and Neck Surgery\u00a0at Harvard Medical School and a CHORD trial investigator. \u201cIn the pivotal trial, the one-time gene therapy demonstrated rapid,\u00a0meaningful and consistent hearing responses, with most\u00a0children achieving remarkable hearing improvements. I\u2019ve witnessed firsthand my trial participant responding to their mother\u2019s voice, dancing to music and interacting with the world, and these moments are now possible for more children born with this specific form of hearing loss.\u201d<\/p>\n

\u201cOtarmeni is a huge scientific leap and is representative of Regeneron\u2019s approaches to continually push the boundaries of science to benefit humanity,\u201d said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer of\u00a0Regeneron. \u201cThis unprecedented breakthrough in gene therapy has already proven to be life-changing for many of the children in our clinical trial and their families. We are honored to be in the position to be the first company to ever offer such a gene therapy advance for free to those in the U.S. and serves to highlight our belief that the biopharmaceutical industry can be a genuine force for good in the world.\u201d<\/p>\n

\n OTOF<\/em>-related hearing loss is an ultra-rare condition, affecting about 50 newborns per year in the U.S. Though all structures within the ear are intact, variants in the OTOF<\/em> 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\u00a0OTOF<\/em>-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.<\/p>\n

\u201cConnection and communication are at the heart of how we experience the world \u2013 whether that happens through listening and spoken language, sign language, the use of technology, or a combination of approaches,\u201d said Janet DesGeorges, Executive Director of Hands & Voices. \u201cFamilies deserve access to balanced information and a range of options when navigating genetic hearing loss. As new treatments and innovations emerge, families can assess available options and choose the approach best suited to their unique circumstances.\u201d<\/p>\n

The FDA approval is based on results from the pivotal CHORD trial, in which 20 participants (aged 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=10). Among the trial participants, efficacy results found:<\/p>\n