FDA OKs First-in-Human Trial for BGTC-Supported Rare Disease Gene Therapy

The Food and Drug Administration on Thursday approved the first gene therapy for a rare inherited form of hearing loss, Regeneron announced. The treatment, called Otarmeni, is designed for people born deaf because of a mutation in the OTOF gene, which normally helps the body make a protein needed to hear. About 50 babies in the U.S. are born with the condition each year. Otarmeni delivers a working copy of the gene directly into the inner ear through a single injection into the cochlea while the patient is under general anesthesia. The FDA approval stemmed from a clinical trial involving 20 children ages 10 months to 16 years. After five months, 16 children showed hearing improvement. Of those followed for at least 11 months, five of 12 had hearing that was essentially normal, including the ability to hear whispers without assistive devices. Researchers reported the treatment was generally safe with no major serious side effects observed. Regeneron said it will provide the treatment at no cost to U.S. patients. The FDA fast-tracked Otarmeni through its Commissioner's National Priority Voucher program, which speeds reviews for products targeting major unmet medical needs. Otarmeni is the first gene therapy approved under the program.