FDA Approves First Genetic Therapy for Deafness

Hearing loss is a condition that affect numerous people around the world, but it can take many forms and have many causes. In some cases, a mutated gene is responsible. One such form is autosomal recessive deafness 9, which is caused by mutations in the OTOF gene. As genetic therapies become more widespread, so too has research into the OTOF gene and whether a treatment can be found to counteract the effects of these mutations.

Now, one treatment in this category has received FDA approval: Otarmeni, which is made by the biotechnology company Regeneron. Approval of Otarmeni was fast-tracked as part of the Commissioner’s National Priority Voucher program, a system designed to expedite the approval of medications that meet certain criteria. It marks both the first gene therapy to be approved under this program and the first FDA-approved genetic therapy to treat deafness.

“Through the national priority voucher pilot program, the agency is accelerating therapies for rare diseases with unmet medical needs while proving we can successfully review even the most complex submissions — such as novel dual vector gene therapies and combination products requiring coordination across multiple offices and centers — in significantly shortened timeframes,” FDA Commissioner Marty Makary said in a statement.

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According to an announcement by Regeneron, the condition that Otarmeni addresses affects approximately 50 newborns in the U.S. each year. The company went on to share that it would make this medication available for free to, as they phrased it, “clinically eligible individuals in the U.S.”

Experts in the field say that this new genetic therapy could represent a significant step forward in treatments for deafness. In comments made to NPR, Mass Eye and Ear‘s Zheng-Yi Chen called the drug’s approval “an historical event, a landmark, a great development for the whole field.”

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Tobias Carroll

Tobias Carroll lives and writes in New York City, and has been covering a wide variety of subjects — including (but not limited to) books, soccer and drinks — for many years. His writing has been published by the likes of the Los Angeles Times, Pitchfork, Literary Hub, Vulture, Punch, the New York Times and Men’s Journal. At InsideHook, he has…