FDA approves first gene therapy for genetic hearing loss
The Food and Drug Administration on Thursday approved Regeneron’s Otarmeni, the first gene therapy for genetic hearing loss.
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The pharmaceutical maker said it would offer the drug 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 United States each year and is caused by a mutation in a gene called OTOF. The approval was granted under the Commissioner’s National Priority Voucher, an FDA pilot program intended to expedite drug review.
“This is truly life-changing for families with hearing-impaired children,” Dr. Eliot Shearer, a pediatric otolaryngologist at Boston Children’s Hospital and principal investigator of the Regeneron trial.
The only other treatment option for children with genetic deafness is cochlear implants, Shearer said, which restore the ability to hear speech and music but reduce the fidelity of sound.
Thanks to gene therapy, enhanced hearing is “available 24/7 and does not require batteries,” he added.
The drug works by replacing the faulty OTOF gene, which is thought to work by giving the body instructions to make a protein called otoferlin. This protein plays a role in transmitting impulses from cochlear cells in the ear to nearby nerves, which transmit signals to the brain. Without otoferlin, sound never reaches the brain.
Thursday’s approval was based on a trial of 20 children diagnosed with the OTOF mutation who received a single dose of treatment in one or both ears.
The trial found it improved hearing in 16 of 20 children, with five children developing the ability to detect whispers. Side effects included middle ear infection or inflammation, vomiting, nausea, and dizziness.
The approval was touted by President Donald Trump during an Oval Office announcement Thursday afternoon regarding a drug pricing deal with Regeneron to provide many of its drugs at a discount on the TrumpRx self-payment platform.
Even though the company provides the drug for free, it has no say in the cost of administering it.
Shearer said the procedure is similar to cochlear implant surgery, where doctors access the inner ear to administer the medication while the patient is under general anesthesia.
The drug is not approved outside the United States, so Regeneron does not have a list price for people abroad who want the treatment. Prices for gene therapy can run into the millions.
Sarah Emond, president and CEO of the Institute for Clinical and Economic Review, a nonprofit group that evaluates the cost and effectiveness of drugs, said Regeneron should be commended for its award.
“Regeneron has shown us that one option we can consider to ensure affordable patient access to these therapies is to not bill the healthcare system for the therapy,” Emond said.
