FDA approves first gene therapy for genetic hearing loss
NBC News • • business
Key Points:
- The FDA approved Regeneron's Otarmeni, the first gene therapy for a rare genetic hearing loss caused by mutations in the OTOF gene, affecting about 50 U.S. babies annually.
- Regeneron will provide Otarmeni free of charge to U.S. patients, although the cost of administering the therapy remains separate and potentially expensive.
- Clinical trials showed that a single dose improved hearing in 16 out of 20 children, with some able to detect whispers, and side effects included middle ear infections and nausea.
- Unlike cochlear implants, Otarmeni offers continuous hearing improvement without reliance on batteries, by replacing the faulty gene responsible for transmitting sound signals to the brain.
- The approval was fast-tracked under an FDA pilot program and highlighted by President Trump, with experts praising Regeneron's decision to forgo charging for the drug to enhance patient access.
