MILWAUKEE — Children’s Wisconsin says it has become the first center in the nation to provide FDA-approved Otarmeni gene therapy for OTOF-related genetic hearing loss outside of a clinical trial, delivering the treatment to two children.
The milestone follows Children’s Wisconsin being designated as one of five “Activated Treatment Centers” in the United States approved to offer the therapy, according to a news release from the health system. The designation is intended for sites with the specialized expertise and multidisciplinary infrastructure required to deliver the treatment.
Otarmeni, developed by Regeneron, is described in the release as 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 is designed to deliver a healthy copy of the OTOF gene directly to the inner ear, addressing the underlying genetic cause of the condition, the release states.
“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,” the release states, describing the hospital’s role in the transition from research to clinical care.
“At Children’s Wisconsin, we don’t simply adopt medical breakthroughs—our teams help make them possible,” Gil Peri, president and CEO of Children’s Wisconsin, said in the release. “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.”
Diagnosing a rare form of hearing loss
Children’s Wisconsin said patient identification is a key part of expanding access to the therapy. The release notes that diagnosing OTOF-related hearing loss requires specialized audiology testing, genetic evaluation and expertise in rare pediatric hearing conditions.
“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 Dr. Brittney Gniedziejko, a pediatric audiologist and ambulatory manager of audiology and clinical research at Children’s Wisconsin.
Access closer to home
Children’s Wisconsin said its Activated Treatment Center designation could mean more Midwestern families have access to the treatment without traveling long distances.
“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 Dr. Michael Harris, chief of otology and neurotologic skull base surgery at the Medical College of Wisconsin and a Children’s Wisconsin co-surgeon on the procedure, according to the release.
Children’s Wisconsin said it offers pediatric hearing care services that include diagnosis, medical and surgical treatment, audiology services and long-term support for children with hearing loss.
Official link
More information is available at: https://childrenswi.org/find-care/ear-nose-and-throat/treatments/otarmeni-gene-therapy
