{"id":591247,"date":"2026-04-24T18:35:00","date_gmt":"2026-04-24T18:35:00","guid":{"rendered":"https:\/\/buglecall.org\/?p=591247"},"modified":"2026-04-24T18:35:00","modified_gmt":"2026-04-24T18:35:00","slug":"fda-approves-first-gene-therapy-to-treat-deafness-2","status":"publish","type":"post","link":"https:\/\/buglecall.org\/?p=591247","title":{"rendered":"FDA Approves First Gene Therapy To Treat Deafness"},"content":{"rendered":"

FDA Approves First Gene Therapy To Treat Deafness<\/span><\/p>\n

\n

Authored by Zachary Stieber via The Epoch Times<\/a> (emphasis ours),<\/em><\/p>\n

Federal regulators on Thursday approved the first gene therapy to restore hearing,<\/strong> just two months after the therapy\u2019s maker formally requested a license.<\/p>\n

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Dr. Marty Makary, the Food and Drug Administration’s commissioner, in an undated file photograph. Madalina Vasiliu\/The Epoch Times<\/em><\/figcaption><\/figure>\n

<\/p>\n

The Food and Drug Administration approved the Regeneron therapy, Otarmeni, <\/strong>for children and adults with severe or profound hearing loss associated with OTOF gene variants.<\/p>\n

The approval came 61 days after Regeneron filed for a biologics license, under a new effort known as the Commissioner\u2019s National Priority Voucher Program that emphasizes quickly reviewing applications for products that address unmet needs.<\/p>\n

\u201cToday\u2019s approval is a significant milestone in the treatment of genetic hearing loss<\/strong>,\u201d Dr. Marty Makary, the FDA\u2019s commissioner, said in a statement.<\/p>\n

\u201cThrough 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\u2014such as novel dual vector gene therapies and combination products requiring coordination across multiple offices and centers\u2014in significantly shortened timeframes.\u201d<\/p>\n

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.<\/p>\n

To treat deafness, Otarmeni is administered to each ear using a syringe and catheter provided in the accompanying kit.<\/p>\n

Regulators approved Regeneron\u2019s 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.<\/p>\n

Common side effects of the drug included ear infection, nausea, and dizziness.<\/strong><\/p>\n

Dr. A. Eliot Shearer, an otolaryngologist at Boston Children\u2019s Hospital and an investigator in the trial, said in a statement released by Regeneron that the one-time therapy provides \u201cremarkable hearing improvements.\u201d<\/p>\n

\u201cI\u2019ve witnessed firsthand my trial participant responding to their mother\u2019s 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,\u201d he said.<\/p>\n

<\/a><\/p>\n

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Dr. Marty Makary, the Food and Drug Administration’s commissioner, in an undated file photograph. Madalina Vasiliu\/The Epoch Times<\/em><\/figcaption><\/figure>\n

<\/p>\n

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

Dr. George D. Yancopoulos, president and chief scientific officer of Regeneron, said in a statement that Otarmeni was an \u201cunprecedented breakthrough\u201d in gene therapy.<\/p>\n

\u201cOtarmeni is a huge scientific leap and is representative of Regeneron\u2019s approaches to continually push the boundaries of science to benefit humanity,\u201d he said.<\/p>\n

\u201cThis 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.\u201d<\/p>\n<\/div>\n

Tyler Durden<\/a><\/span>
\nFri, 04\/24\/2026 – 14:35<\/span><\/p>","protected":false},"excerpt":{"rendered":"

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\u2019s maker formally requested a license. Dr. Marty Makary, the Food and Drug Administration’s commissioner, in an undated file photograph.… Continue reading FDA Approves First Gene Therapy To Treat Deafness<\/span><\/a><\/p>\n","protected":false},"author":1,"featured_media":591241,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"rop_custom_images_group":[],"rop_custom_messages_group":[],"rop_publish_now":"initial","rop_publish_now_accounts":[],"rop_publish_now_history":[],"rop_publish_now_status":"pending","footnotes":""},"categories":[18,19,10,21,12,11,9],"tags":[],"class_list":["post-591247","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-cancel-culture","category-censorship","category-civil-liberties","category-election-integrity","category-equal-justice","category-free-speech","category-religious-freedom","entry"],"_links":{"self":[{"href":"https:\/\/buglecall.org\/index.php?rest_route=\/wp\/v2\/posts\/591247","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/buglecall.org\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/buglecall.org\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/buglecall.org\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/buglecall.org\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=591247"}],"version-history":[{"count":0,"href":"https:\/\/buglecall.org\/index.php?rest_route=\/wp\/v2\/posts\/591247\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/buglecall.org\/index.php?rest_route=\/wp\/v2\/media\/591241"}],"wp:attachment":[{"href":"https:\/\/buglecall.org\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=591247"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/buglecall.org\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=591247"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/buglecall.org\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=591247"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}