
The Food and Drug Administration on Thursday approved Regeneron’s Otarmeni, the first gene therapy for genetic hearing loss. Subscribe to read this story ad-free Get unlimited access to ad-free articles and exclusive content. The drugmaker said it will offer the drug for free to U.S. patients. Otarmeni is approved to treat a very rare form of hearing loss that affects about 50 babies born in the U.S. each year and is caused by a mutation in a gene called OTOF.
Main Idea: Regeneron won FDA approval for the first gene therapy for a rare genetic hearing loss, and the company says it will provide the treatment free to U.S. patients.
Key Points:
The gene therapy is only for a very rare hearing loss, so most US families will not benefit and the hospital procedure may still carry side effects.
Regeneron’s free gene therapy could help a few newborns hear better without cochlear implants, and FDA approval may speed future treatments for rare diseases.
Rate how each entity in this article affected the American people.
Central company behind the approved gene therapy and the pricing/access decision.
The regulator that approved the first gene therapy for this genetic hearing-loss condition.
Named political figure who publicly touted the approval during the Oval Office announcement.
Named hospital where the quoted pediatric otolaryngologist works and where trial leadership is based.
Named clinician and trial investigator quoted about the therapy’s effects and procedure.
Named nonprofit whose president commented on the pricing model.
Named nonprofit executive quoted on the therapy’s pricing and access approach.
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