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

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 Trial
The 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 expansion cohort (Part B), participants receive Otarmeni in both ears at the selected dose from Part A.

Hearing improvements were assessed by average pure tone audiometry (PTA) and auditory brainstem response (ABR). PTA is the gold standard measurement of hearing sensitivity and is measured through behavioral responses to sound (e.g., turning head towards sound) that is emitted at different intensity levels and measured in decibels (dB). ABR complements these behavioral responses, serving as an objective measure of synchronized neural response, to sound. At baseline, all participants had profound hearing loss (behavioral PTA), and no electrophysiological (ABR) responses at maximum sound levels.

About Otarmeni™ (lunsotogene parvec)
Otarmeni is an in vivo dual adeno-associated virus serotype 1 (AAV1) vector-based gene therapy designed to restore durable, physiological hearing to individuals by delivering a working copy of the OTOF gene through a modified, non-pathogenic virus that is delivered via an infusion into the cochlea under general anesthesia (similar to the procedure used for cochlear implantation). In this gene therapy, the newly introduced OTOF gene is under the control of a proprietary cell-specific Myo15 promoter, which is intended to restrict expression only to hair cells that normally express the otoferlin protein.

Otarmeni is currently approved in the U.S. 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 gene, preserved outer hair cell function, and no prior cochlear implant in the same ear.

IMPORTANT SAFETY INFORMATION FOR U.S. PATIENTS

What is the most important information to know about OTARMENI?

Before receiving OTARMENI:

• it is recommended to receive age-appropriate vaccinations, at least 1 month before the first corticosteroid dose and at least 1 month after the last corticosteroid dose.
• consult with your healthcare provider and surgeon regarding vaccination status against meningitis, since meningitis is a known risk of inner ear surgery.
• pregnancy status should be verified (if you are sexually active and able to become pregnant).
  
  

The following serious side effects may occur with the surgery required to administer OTARMENI:

• Vertigo, ringing in ear(s), cerebral spinal fluid leak, partial facial paralysis or weakness, change in taste, meningitis, wound infection, serious infection of the bone behind the ear (mastoiditis), numbness around the ear, blood or fluid collection at surgical site, and inflammation of the inner ear.
  
  

The most common side effects that may occur with OTARMENI include middle ear infection, vomiting, nausea, dizziness, procedural pain, walking disturbance, and rapid involuntary eye movements.

Other clinically significant side effects, each occurring in 1 person in the clinical study, included temporary balance disorder, abnormal otoacoustic emissions, and wound separation.

Because small quantities of OTARMENI may be present in bodily fluids/waste, any materials that may be contaminated should be placed in a sealable bag and disposed of into regular trash for the first two weeks following administration of OTARMENI. Practice proper hand hygiene, such as hand washing, when coming into direct contact with bodily fluids/waste.

Talk to your healthcare provider for medical advice or any questions about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. You may also report side effects to Regeneron Pharmaceuticals at 1‑866‑500‑GENE (1‑866‑500‑4363).

Please see accompanying full Prescribing Information.

About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases.

Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite^®, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center^® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases.

For more information, please visit www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X.

Forward-Looking Statements and Use of Digital Media
This press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. (“Regeneron” or the “Company”), and actual events or results may differ materially from these forward-looking statements. Words such as “anticipate,” “expect,” “intend,” “plan,” “believe,” “seek,” “estimate,” variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Products”) and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, “Regeneron’s Product Candidates”) and research and clinical programs now underway or planned, including without limitation Otarmeni™ (lunsotogene), an in vivo adeno-associated virus vector-based gene therapy; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new indications for Regeneron’s Products, such as Otarmeni for the treatment of biallelic OTOF variant-associated hearing loss in the European Union; uncertainty of the utilization, market acceptance, and/or commercial success of Regeneron’s Products and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies discussed or referenced in this press release, on any of the foregoing or any potential regulatory approval of Regeneron’s Products and Regeneron’s Product Candidates (such as Otarmeni); the ability of Regeneron’s collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron’s Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates and risks associated with tariffs and other trade restrictions; safety issues resulting from the administration of Regeneron’s Products and Regeneron’s Product Candidates (such as Otarmeni) in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron’s Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize Regeneron’s Products and Regeneron’s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement or copay assistance for Regeneron’s Products from third-party payors and other third parties, including private payor healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payors and other third parties and new policies and procedures adopted by such payors and other third parties; changes to drug pricing regulations and requirements and Regeneron’s pricing strategy, including in connection with Regeneron’s April 2026 agreements with the U.S. government; other changes in laws, regulations, and policies affecting the healthcare industry; competing products and product candidates (including biosimilar products) that may be superior to, or more cost effective than, Regeneron’s Products and Regeneron’s Product Candidates; the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron’s agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable), to be cancelled or terminated; the impact of public health outbreaks, epidemics, or pandemics on Regeneron's business; and risks associated with litigation and other proceedings and government investigations relating to the Company and/or its operations (including the pending civil proceedings initiated or joined by the U.S. Department of Justice and the U.S. Attorney's Office for the District of Massachusetts), risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA® (aflibercept) Injection), the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron’s business, prospects, operating results, and financial condition. 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Contacts:
Media Relations         Tammy Allen Tel: +1 914-306-2698 tammy.allen@regeneron.com	Investor Relations Mark Hudson Tel: +1 914-847-3482 mark.hudson@regeneron.com

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