WTAS: FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss Under National Priority Voucher Program

Groundbreaking AAV-based gene therapy offers potential treatment for patients with OTOF gene-associated severe-to-profound and profound hearing loss

The U.S. Food and Drug Administration on April 23, 2026, approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy. Otarmeni is indicated for the treatment of pediatric and adult patients with severe-to-profound and profound sensorineural hearing loss (any frequency >90 dB HL) associated with molecularly confirmed biallelic variants in the OTOF gene.

Following the publication of powerful results of hearing restoration in the New England Journal of Medicine, the FDA acted swiftly to grant a national priority voucher for an accelerated review. Today’s approval was issued 61 days after BLA filing, marking the sixth approval under the Commissioner's National Priority Voucher (CNPV) pilot program and the first gene therapy product approved under the program. It is also tied for the fastest BLA approval in modern FDA history.

Headlines
Medical Professionals
Advocates & Organizations
Influencers & Members of the Media
 

Headlines:

• The New York Times: New Gene Therapy Enables Children With a Rare Form of Deafness to Hear
• The Wall Street Journal: FDA Approves First-Ever Gene Therapy to Restore Hearing
• NBC News: FDA approves first gene therapy for genetic hearing loss
• Fox News: BioPharma Dive: FDA approves Regeneron’s hearing loss gene therapy
• CNN: FDA approves first gene therapy for inherited deafness, shown to restore hearing for children with rare condition
• Bloomberg: Regeneron to Offer Hearing-Loss Therapy for Free After FDA Nod
• NPR: The FDA gives the green light to the first gene therapy for deafness
• Reuters: Regeneron wins FDA approval for first gene therapy for genetic hearing loss
• Forbes: New Gene Therapy For Rare Hearing Loss Wins FDA Approval—What To Know
• New York Post: FDA OKs ‘life-changing’ gene therapy for hearing loss — and it’s free
• CNBC [CLIP]: Regeneron CEO on first-ever FDA approved gene therapy to restore hearing
• Endpoints News: Regeneron snags hearing loss gene therapy approval, closes US pricing deal
• Fierce Pharma: Regeneron ushers in new genetic medicine era with groundbreaking gene therapy approval
• US News & World Report: First Gene Therapy for Genetic Hearing Loss Gains FDA Approval
• BioPharma Dive: FDA approves Regeneron’s hearing loss gene therapy
• Medical Professionals Reference: Otarmeni Wins FDA Approval for Genetic Sensorineural Hearing Loss
• MedWatch: FDA approves Regeneron gene therapy for genetic hearing loss
• Nature Biotechnology: Historic FDA approval brings first gene therapy for genetic deafness
• The Hearing Review: FDA Grants Accelerated Approval to Otarmeni Gene Therapy for OTOF-Related Hearing Loss
• Becker’s Hospital Review: FDA approves 1st gene therapy for genetic hearing loss
• Pharmacy Times: FDA Grants Accelerated Approval to Otarmeni, First and Only Gene Therapy for Genetic Hearing Loss
• Precision Medicine Online: FDA Approves First Gene Therapy for Rare Form of Genetic Hearing Loss
• Hearing Health Matters: Regeneron Secures Landmark FDA Approval for Hearing Loss Gene Therapy, Otarmeni

Medical Professionals:

• Columbia University Irving Medical Center Hearing Specialist, Lawrence Lustig, via The Wall Street Journal: “In my wildest dreams I never thought we’d be here in my lifetime…”
• University of California, San Francisco Pediatric Otolaryngologist, Dr. Dylan Chan, via The New York Times: “…[G]roundbreaking…This is the first time in history that there has been a medical therapy that has enabled deaf children to hear.”
• Boston Children’s Hospital Pediatric Otolaryngologist and Principal Investigator on the Regeneron trial, Dr. Eliot Shearer, via NBC News: “This really is life-changing for families with children with hearing loss…” With gene therapy, the improved hearing is “on 24/7 and doesn’t rely on batteries…”
• Boston Children’s Hospital Pediatric Otolaryngologist and Principal Investigator on the Regeneron trial, Dr. Eliot Shearer, via CNN: “It’s not like a little incremental change, which I think sometimes in science, things can feel like…It’s completely life-changing.”
• Boston Children’s Hospital pediatric otolaryngologist and principal investigator on the Regeneron trial, Dr. Eliot Shearer, via BioPharma Dive: “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… a new era in the treatment of genetic forms of hearing loss, where reinstating 24/7 natural hearing is now possible…”
• Massachusetts Eye and Ear Infirmary Director of Pediatric Otology and Neurotology, Dr. Daniel Lee, via The New York Times: “We have now entered the era of biological treatment for inner ear hearing loss…”
• Massachusetts Eye and Ear Infirmary Associate Scientist, Zheng-Yi Chen, via NPR: "It's the first time in history there's a new drug for hearing loss…I think it's an historical event, a landmark, a great development for the whole field…”
• Hear.com Senior Audiologist, Rachel Artsma, via Fox News: “[A] major milestone in hearing health…While this one‑time gene therapy is limited to a specific genetic population, it underscores the importance of early diagnosis and genetic testing… Advances like this reflect meaningful progress in hearing science, and demonstrate how biologic treatments, technology and clinical care are increasingly converging to improve long‑term outcomes for patients.”
• Regeneron Co-founder and President, Dr. George Yancopoulos, via CNN: “We want to make an example of how science, and in this case biotech, can really deliver a gift to people – in this case, the gift of hearing…”
• Regeneron Co-founder and President, Dr. George Yancopoulos, via Regeneron press release: “Otarmeni is a huge scientific leap and is representative of Regeneron’s approaches to continually push the boundaries of science to benefit humanity…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.”

Advocates & Organizations:

• Sierra Smith, mother of a clinical trial participant via NPR: “It's incredible. Now he can hear me tell him how much I love him…And he knows has a name now. And he's discovering all these different sounds…It's so mind-blowing. I feel like I'm so blessed and I'm the luckiest mom on earth.”
• Sierra Smith, mother of a son with deafness treated with Regeneron via Reuters: "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…”
• Kerri, mother of a clinical trial participant via CNN: “It’s miraculous… You go from being told your child’s profoundly deaf and may only ever hear with technology to your child’s hearing right alongside his friends. … This is just amazing.”
• Kerri, mother of a clinical trial participant via The New York Times: “[C]ompletely changed our lives…We are so fortunate…Our baby was born deaf, and now he can hear.”
• Regeneron CEO, Leonard Schleifer, via CNBC: “This is the first time ever that a gene that has replaced something and restored one of our senses, and you realize how important hearing is…Everybody came together on this one: our scientists, the patients, the doctors, the FDA. The commissioner’s voucher. People have been making comments about the FDA, that they’re lowering the standards. I guarantee you they’re not. We had so much up and back on this. They’re keeping the standards high, but this program says if there’s an important program, let’s move it along fast. I say kudos to the agency on that.”
• Regeneron’s Vice President of Genetic Medicines, Jonathan Whitton, via BioPharma Dive: “Is there really any difference between a 16- and a 21-year-old for something like this? We don’t think so…It’s a pleasant surprise for a lot of people in our field, me included, that it looks like the treatment window is so broad.”
• Regeneron’s Vice President of Genetic Medicines, Jonathan Whitton, via NPR: "Now for the first time we are talking about medicines that actually enable the ear to hear. It's the beginning of a new era, honestly…These are incredible results for somebody like me who been in this field for a couple of decades. It's something I hadn't imagined only a few years ago could be possible…What we've seen has been quite remarkable."
• Regeneron Genetic Medicines Co-Head, Aris Baras, via Endpoints News: “A child that’s born deaf and then in a quite short amount of time has the possibility to regain normal hearing. It’s absolutely remarkable…This is the first of what we hope could be lots of opportunities in hearing loss.”
• MAHA Action (@MAHA_Action) via X: “President Trump just announced the FDA has approved a treatment that cures a disease that causes deafness. The company Regeneron has agreed to provide the treatment free of charge. This breakthrough is already allowing children who were born unable to hear to hear for the first time. A major step forward for families affected by a condition that until now was considered untreatable.”
• Institute for Clinical and Economic Review President and CEO, Sarah Emond, via NBC News: “Regeneron has shown us that one option we can consider that will ensure affordable access for patients to these therapies is to not charge the health system for the therapy…”
• Hands & Voices Executive Director via Regeneron press release: “Connection and communication are at the heart of how we experience the world – whether that happens through listening and spoken language, sign language, the use of technology, or a combination of approaches…Families deserve access to balanced information and a range of options when navigating genetic hearing loss. As new treatments and innovations emerge, families can assess available options and choose the approach best suited to their unique circumstances.”
• Hearing Health Foundation (@HearingHealthFn) via X: “The surgical procedure to administer Otarmeni uses an approach similar to cochlear implantation and allows use in young infants.... Regeneron will provide Otarmeni at no cost to clinically eligible individuals in the U.S.”
• Patients for Affordable Drugs (@P4AD_) CEO, Merith Basey, via X: “Today’s approval of Otarmeni is a landmark moment, and we are thrilled for the families who will be able to access this therapy for free…”

Influencers & Members of the Media:

• World News Tonight via X: “The FDA has approved the first-ever medical treatment that allows deaf children with a rare genetic condition to hear. Regeneron says it will offer the new gene therapy free of charge to the small pool of eligible patients. @DavidMuir reports.”
• Becky Quick, Host of Squawk Box, via CNBC: “This is really pretty amazing.”
• Greta Van Susteren, Host of The Record With Greta Van Susteren, via Newsmax: “That’s awesome. Very exciting news out of the FDA.”
• Grant Stinchfield, Host of Stinchfield Tonight, via Real America’s Voice: “So President Trump held a fabulous news conference with RFK Jr., making a deal with Regeneron to do some unbelievable things, not just to keep prescription medication prices low, which that was part of it, but he's got a deal now for a treatment for these rare genetic diseases that literally cure deafness in a young boy who had been deaf since birth. This is fabulous and they're going to offer this to American citizens for nothing.”
• Batya Ungar-Sargon, Host of Batya, via NewsNation: “Many of the great wins are being eclipsed, and that happened again this week…The FDA also approved a new drug from Regeneron – a gene therapy that can cure deafness in children. The press conference announcing the deal included one tiny beneficiary. In any other administration, curing children of deafness so they can hear their momma say ‘I love you’ would have been the biggest story of the week. And we just couldn’t let it slide without giving it it’s due.”
• CBS Mornings via Facebook: “‘Let Travis be Travis’: Two-year-old Travis Smith is a no shoes kind of guy, his mom, Sierra Smith, told CBS Mornings, after the toddler played in the Oval Office Thursday while President Trump spoke. Sierra and Travis were at the White House, as the president spoke about the biotech company Regeneron, which ran a clinical trial for an experimental therapy that Travis, who was born deaf, took part in.”
• Steve Shultz (@elijahststeve) via X: “Wow. UNPRECEDENTED!! FREE, first-of-its kind gene therapy for all children with hearing loss. This should be shouted FROM THE HOUSETOPS by the media.  Whichever major media outlet refuses to cover this story for your children... well, they are saying "WE DON'T CARE ABOUT THIS BREAKTHRU FOR YOUR CHILD!"”
• Niko McCarty (@NikoMcCarty) via X: “Huge news. A gene therapy for deafness, targeting a mutation in the OTOF gene, is approved. Restored hearing in 9 of 12 children, well enough that they could stop using cochlear implants. The treatment will be free in the United States.”
• Martin Varsavsky (@martinvars) via X: “From science fiction to clinic. A gene therapy that restores hearing in children born deaf is now an approved drug. Biotech delivers when regulators let it. Europe watches from the sidelines while it legislates AI.”
• Eric Daugherty (@EricLDaugh) via X: “WOW! President Trump just welcomed 2YO Travis Smith to the Oval who was one of the FIRST children treated with a gene therapy that CURED his hearing loss  Which is now backed by the FDA. "He can listen to music. And he loves it.”  Incredible!”
• Nick Sortor (@nicksortor) via X: A BEAUTIFUL moment just occurred in the Oval Office. President Trump just told the mother of 2-year-old Travis the FDA has APPROVED a therapy that CURED his deafness. TRUMP: "He took this miracle cure and now he can hear his mom, Sierra, say 'I love you.'" MOM: "He didn't know his name. He couldn't hear me tell him how much I love him. And now with this amazing surgery, he can listen to music. He loves it and he loves to dance and he loves instruments, and I'm so proud of him and he's been so brave through all of this. Now he can hear, and he has a bright future ahead of him. I'm so thankful...
• Just The News (@JustTheNews) via X: “"The FDA has just approved a new drug from Regeneron called Otarmony, a gene therapy curing a rare disease that causes deafness...he took this miracle cure, and now he can hear his mom." @POTUS is overjoyed to hear that breakthrough technology Otarmony has allowed a previously deaf toddler to hear.”
• Wall Street Apes (@WallStreetApes) via X: “President Donald Trump announces an agreement with the pharmaceutical company Regeneron. The FDA also approved a new drug from Regeneron, a gene therapy that can cure deafness in children. “Regeneron committed to slashing the cost of its meds and giving American patients most favored nation pricing as part of the deal”. One price cut example is Regeneron’s cholesterol drug Praluent (alirocumab) will see a it’s drops significantly, from $537 to $225. Regeneron will also provide its newly approved hearing-loss treatment Otarmeni (lunsotogene parvec-cwha) at no cost to eligible US patients. Huge win.”
• Gemini (@Gemini) via X: “JUST IN: FDA approves first ever gene therapy that restores hearing in children born deaf, with treatment available at no cost”