Children’s Wisconsin First in the Nation To Provide FDA-Approved Gene Therapy for Treatment of Genetic Hearing Loss

The achievement comes shortly after Children's Wisconsin was designated as one of only five Activated Treatment Centers in the United States approved to offer the therapy. The designation recognizes the specialized expertise and multidisciplinary infrastructure required to safely deliver the treatment.

As one of the leading enrollment sites in the Otarmeni clinical trial, Children's Wisconsin cared for more children receiving the investigational therapy than nearly any other participating center, helping pave the way for its transition from research to clinical care.

"At Children's Wisconsin, we don't simply adopt medical breakthroughs - our teams help make them possible," said Gil Peri, President and CEO of Children's Wisconsin. "From helping lead the clinical research to becoming the first center in the nation to provide this therapy outside of a clinical trial, this milestone reflects our commitment to innovation and to bringing life-changing treatments to children and families."

Otarmeni, developed by Regeneron, is the first FDA-approved gene therapy for otoferlin gene (OTOF)-related hearing loss, a rare genetic condition that prevents sound signals from being properly transmitted from the inner ear to the brain. The therapy delivers a healthy copy of the OTOF gene directly to the inner ear, addressing the underlying genetic cause of hearing loss.

Children's Wisconsin plays a critical role not only in delivering the therapy, but also in identifying patients who may benefit from it. Diagnosing OTOF-related hearing loss requires specialized audiologic testing, genetic evaluation and expertise in rare forms of pediatric hearing loss.

"Despite being a rare condition, OTOF-related hearing loss is one that the pediatric audiologists at Children's Wisconsin have extensive expertise in diagnosing and managing," said Brittney Gniedziejko, Aud, Pediatric Audiologist and Ambulatory Manager of Audiology and Clinical Research Unit at Children's Wisconsin. "Together with our partners in otology, we are now able to provide an exciting new treatment option for patients and families. This achievement demonstrates the specialized expertise and forward-thinking care that Children's Wisconsin brings to pediatric hearing care every day."

For children and families across Wisconsin and the Midwest, becoming an Activated Treatment Center means access to one of the most advanced treatments available for genetic hearing loss close to home.

"This accomplishment is about more than being first. It's about giving children and families access to a treatment that wasn't possible a few years ago," said Michael Harris, MD, Chief of Otology and Neurotologic Skull Base Surgery at the Medical College of Wisconsin and the Children's Wisconsin co-surgeon on the procedure. "Delivering a first-of-its-kind therapy requires precision, preparation and an exceptional multidisciplinary team. We're proud to have helped bring this treatment into clinical practice and excited about what it means for the future of hearing care."

Children's Wisconsin has long been recognized as a leader in pediatric hearing care, offering comprehensive diagnosis, medical and surgical treatment, audiology services and long-term support for children with hearing loss. As gene therapies continue to reshape the future of pediatric medicine, Children's Wisconsin is committed to ensuring children and families have access to the latest advances through the expertise of nationally recognized specialists, leading-edge research and multidisciplinary care.

Click here for more information on OTOF-related hearing loss and treatment.