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The U.S. Food and Drug Administration has approved Regeneron’s gene therapy for a rare genetic form of deafness, the company said on Thursday.
This approval, granted under the FDA’s new priority voucher program, marks the introduction of the first gene therapy for genetic hearing loss to the market.
Regeneron said the therapy Otarmeni will be available for free to patients in the United States. Regeneron is also set to announce a drug pricing deal with the White House this afternoon.
The gene therapy targets otoferlin-related hearing loss, a condition caused by variants in the OTOF gene that affects 20–50 newborns in the U.S. each year.
Otoferlin is a critical protein in the ear’s inner hair cells, essential for transmitting sound signals to the brain.
The therapy delivers a working copy of the OTOF gene to replace the non-functional otoferlin protein using a modified, non-pathogenic virus, delivered via an infusion into the cochlea — a bony cavity within the inner ear.
China’s Refreshgene and Eli Lilly have also been studying therapies for the condition that have restored hearing in children in...