The U.S. Food and Drug Administration (FDA) has officially approved the first gene therapy for the treatment of a rare form of hereditary hearing loss. Medical experts evaluate this as a significant breakthrough, ushering in a brand-new era in the treatment of genetic hearing disorders.
According to statistics, 2 to 3 out of every 1,000 children in the U.S. are born with hearing loss, more than half of which is caused by genetic mutations. Developed by the American biotechnology company Regeneron, the innovative drug Otarmeni aims to treat a rare genetic condition diagnosed in approximately 50 newborns annually in the United States.
The therapy is intended for children and adults with severe-to-profound hearing loss caused by mutations in the OTOF gene. This specific gene encodes a critically important protein responsible for transmitting auditory signals from the inner ear to the brain. The procedure is performed by a surgeon as a one-time injection directly into the ear.
A clinical trial involving 20 pediatric patients ranging from 10 months to 16 years of age demonstrated high efficacy: at least 80% of patients showed significant hearing improvement within a few months.
The economic aspect of the drug is also noteworthy. Generally, gene therapies are extremely expensive, with prices in the U.S. often reaching several million dollars. However, according to a statement by Regeneron, they plan to make this treatment available completely free of charge to American patients who meet the relevant medical criteria.
