FDA approves Otarmeni, first gene therapy for rare OTOF-related infant hearing loss
TL;DR
FDA approves Regeneron's Otarmeni, the first gene therapy for a rare OTOF-mutation–driven hearing loss in infants; in a trial, a single-dose inner-ear injection improved hearing in 16 of 20 children, with some able to detect whispers; the company will provide the drug for free to U.S. patients, while pricing outside the U.S. has not been determined since it is not approved there.
- FDA approves first gene therapy for genetic hearing loss NBC News
- New Gene Therapy Enables Children With a Rare Form of Deafness to Hear The New York Times
- Regeneron ushers in new era with inaugural gene therapy approval Fierce Pharma
- Regeneron to Offer Hearing-Loss Therapy for Free After FDA Nod Bloomberg.com
- The FDA gives the green light to the first gene therapy for deafness NPR
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