FDA Approves First Gene Therapy To Treat Deafness

Authored by Zachary Stieber via The Epoch Times (emphasis ours),

Federal regulators on Thursday approved the first gene therapy to restore hearing, just two months after the therapy’s maker formally requested a license.

The Food and Drug Administration approved the Regeneron therapy, Otarmeni, for children and adults with severe or profound hearing loss associated with OTOF gene variants.

The approval came 61 days after Regeneron filed for a biologics license, under a new effort known as the Commissioner’s National Priority Voucher Program that emphasizes quickly reviewing applications for products that address unmet needs.

“Today’s approval is a significant milestone in the treatment of genetic hearing loss,” Dr. Marty Makary, the FDA’s commissioner, said in a statement.

“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.”

Genetic mutations account for about half of cases of inherited hearing loss. Variants in the OTOF gene account for 2 to 8 percent of those cases.

To treat deafness, Otarmeni is administered to each ear using a syringe and catheter provided in the accompanying kit.

Regulators approved Regeneron’s therapy after reviewing data from a clinical trial involving 24 patients aged 10 months to 16 years. Of the 20 patients included in the efficacy analysis, 80 percent experienced improvement in hearing after receiving Otarmeni.

Common side effects of the drug included ear infection, nausea, and dizziness.

Dr. A. Eliot Shearer, an otolaryngologist at Boston Children’s Hospital and an investigator in the trial, said in a statement released by Regeneron that the one-time therapy provides “remarkable hearing improvements.”

“I’ve witnessed firsthand my trial participant responding to their mother’s voice, dancing to music and interacting with the world, and these moments are now possible for more children born with this specific form of hearing loss,” he said.

Regeneron said it will provide the therapy free of charge to patients in the United States as part of a deal struck with President Donald Trump and announced on Thursday.

Dr. George D. Yancopoulos, president and chief scientific officer of Regeneron, said in a statement that Otarmeni was an “unprecedented breakthrough” in gene therapy.

“Otarmeni is a huge scientific leap and is representative of Regeneron’s approaches to continually push the boundaries of science to benefit humanity,” he said.

“This unprecedented breakthrough in gene therapy has already proven to be life-changing for many of the children in our clinical trial and their families. We are honored to be in the position to be the first company to ever offer such a gene therapy advance for free to those in the U.S. and serves to highlight our belief that the biopharmaceutical industry can be a genuine force for good in the world.”