By Sriparna Roy
April 23 (Reuters) – 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 early trials.
“I’d say for the field, it means a new era and thinking about treatments for hearing loss – this is just the beginning,” said Jonathon Whitton, vice President, auditory global program head at Regeneron.
Travis Smith, born six weeks early and diagnosed with the OTOF mutation, received Regeneron’s experimental therapy at 18 months as part of a clinical trial.
“Watching him be able to interact with other children and even him knowing his name now and turning when I say his name is the craziest thing,” said two-and-a-half-year-old Travis’ mother Sierra, whom Reuters contacted through Regeneron.
“I can tell him how much I love him – it’s such a different world.”
(Reporting by Sriparna Roy, Christy Santhosh and Puyaan Singh in Bengaluru; Editing by Vijay Kishore)
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