Milwaukee-based Children’s Wisconsin became the first center in the U.S. to administer Otarmeni, an FDA-approved gene therapy for genetic hearing loss, outside a clinical trial. The health system treated two children with the therapy.
The health system is one of only five activated treatment centers in the U.S. approved to offer the therapy, according to an Aug. 19 news release. Children’s Wisconsin also participated in the Otarmeni clinical trial.
Developed by Regeneron, Otarmeni is the first FDA-approved gene therapy for hearing loss caused by mutations in the OTOF gene. The one-time treatment delivers a functional copy of the OTOF gene directly to the inner ear.
The FDA approved Otarmeni April 23 for children and adults with severe-to-profound hearing loss caused by OTOF gene mutations. In a clinical trial of 24 pediatric patients, 80% of evaluable patients had improved hearing.
