The European Medicines Agency accepted the marketing application for Otarmeni, a gene therapy targeting rare inherited hearing loss. The company received the filing acceptance under the agency’s Accelerated Assessment pathway, which supports medicines addressing serious unmet medical needs.
Otarmeni, also known as lunsotogene parvec, is designed to treat hearing loss caused by biallelic OTOF gene variants. The therapy uses an adeno-associated virus vector to deliver a functioning copy of the OTOF gene directly into the cochlea. Scientists believe this process may restore hearing by enabling proper communication between sensory cells and auditory nerves.
The EMA filing acceptance marks another regulatory milestone for Otarmeni after its recent approval in the United States. In April 2026, the U.S. Food and Drug Administration granted accelerated approval to Otarmeni for severe-to-profound OTOF-related hearing loss. The therapy became the first approved gene therapy for inherited hearing loss in the country.
Otarmeni previously received orphan designation from the EMA because the condition affects a very limited patient population. Approximately 46 newborns annually in the European Union develop OTOF-related hearing loss. The disorder remains classified as ultra-rare, although it creates lifelong communication challenges for affected children and families.
The EMA application for Otarmeni relies on results from the ongoing CHORD clinical trial. Researchers enrolled infants, children, and adolescents between 10 months and 16 years old in the study. Participants received a single intracochlear infusion either in one ear or both ears. The company evaluated safety, tolerability, and hearing improvement after treatment.
The CHORD trial included 24 participants at the time of the EMA filing. Earlier findings from 20 participants also supported the FDA approval decision for Otarmeni earlier this year. Researchers observed meaningful hearing improvements among treated patients, strengthening confidence in the therapy’s long-term clinical potential.
Otarmeni works differently from conventional hearing devices because it targets the genetic cause of hearing impairment instead of amplifying external sounds. Historically, individuals with OTOF-related hearing loss relied on hearing aids or cochlear implants throughout life. However, these devices cannot fully restore natural sound perception or physiological hearing function.
The company explained that OTOF gene variants prevent the production of functional otoferlin protein. This protein plays a critical role in transmitting sound signals from the inner ear to the auditory nerves. Although ear structures remain intact, patients cannot process sound normally because the signaling pathway fails without sufficient otoferlin activity.
Otarmeni introduces a functioning OTOF gene through a modified non-pathogenic virus. Surgeons administer the therapy directly into the cochlea under general anesthesia during a procedure resembling cochlear implantation. The treatment involves a unique promoter to reduce gene activity only in certain cells in the ear.
Additionally, the EMA granted Accelerated Assessment status to Otarmeni because regulators considered the therapy clinically significant for public health. This review process may shorten evaluation timelines for innovative medicines addressing severe conditions lacking effective treatment options. The designation could help European patients access the therapy more quickly if regulators approve the application.
The CHORD study continues to progress in different countries, such as the US, UK, Spain, Germany, and Japan. Other regulatory filings are planned by the company as it continues to increase its development activities for Otarmeni worldwide. The safety profile for Otarmeni includes risks associated with inner ear surgery and gene therapy administration. These adverse effects include vertigo, nausea, infection of the middle ear, difficulty in walking, and procedural pain. The other effects were balance problems and otoacoustic emissions during clinical evaluation.
Despite these concerns, investigators observed positive results regarding efficacy in the whole clinical trial program. Earlier company disclosures suggested that many people who had been treated experienced hearing improvement after taking Otarmeni. Many of the participants reached hearing capacities that were almost normal in their follow-up period.
Industry experts continue monitoring Otarmeni closely because the therapy represents a major advancement in genetic medicine and sensory restoration. This treatment shows the capability of gene therapy platforms in treating conditions that had been deemed irreversible for all of medicine's history. Regulatory decisions in Europe could therefore influence future investment and research within inherited hearing loss treatment programs.
