{"id":36193,"date":"2026-09-25T17:34:00","date_gmt":"2026-09-25T12:04:00","guid":{"rendered":"https:\/\/www.delveinsight.com\/blog\/?p=36193"},"modified":"2026-09-25T09:35:28","modified_gmt":"2026-09-25T04:05:28","slug":"fayuvi-approval-for-sanfilippo-syndrome","status":"publish","type":"post","link":"https:\/\/www.delveinsight.com\/blog\/fayuvi-approval-for-sanfilippo-syndrome","title":{"rendered":"FAYUVI\u2019s Comeback Story: How Ultragenyx Turned Rejection Into FDA Approval"},"content":{"rendered":"\n<p><strong><em>Summary<\/em><\/strong><\/p>\n\n\n\n<ul class=\"wp-block-list\">\n<li><em>On September 17, 2026, the FDA approved Ultragenyx\u2019s FAYUVI (rebisufligene etisparvovec-hopf) as the first treatment targeting the underlying cause of Sanfilippo syndrome type A in pediatric patients with preserved neurodevelopmental function.<\/em><\/li>\n\n\n\n<li><em>Data from the pivotal Transpher A study and long-term follow-up showed sustained reductions in cerebrospinal fluid heparan sulfate and higher cognitive scores versus an external natural-history cohort.<\/em><\/li>\n\n\n\n<li><em>FAYUVI was rejected by the FDA in July 2025 because of manufacturing issues involving Ultragenyx\u2019s facility and a third-party site. After resubmission and priority review, the therapy received full approval in September 2026.<\/em><\/li>\n\n\n\n<li><em>Ultragenyx priced FAYUVI at a $3.95 million wholesale acquisition cost, making it one of the most expensive therapies ever launched and placing it just below Orchard Therapeutics\u2019 $4.25 million LENMELDY.<\/em><\/li>\n<\/ul>\n\n\n\n<p>For decades, a Sanfilippo syndrome type A diagnosis meant families could only manage symptoms. On September 17, 2026, that changed. The U.S. FDA approved <strong>Ultragenyx&#8217;s FAYUVI (rebisufligene etisparvovec-hopf)<\/strong>, the first treatment for children with MPS IIIA that targets the underlying disease. The approval comes after a 2025 rejection, a record price tag, and a turbulent stretch for <a href=\"https:\/\/www.delveinsight.com\/blog\/ultra-rare-disease-drug-approvals\">rare disease drug approval<\/a> regulation. FAYUVI, formerly known as UX111, is a one-time intravenous gene therapy. It is indicated for the neurologic manifestations of MPS IIIA (Sanfilippo syndrome type A) in pediatric patients with preserved neurodevelopmental function.&nbsp;<\/p>\n\n\n\n<p>The FDA granted standard full approval, not accelerated approval. The therapy uses an AAV9 viral vector to deliver a working copy of the SGSH gene into a patient&#8217;s cells. That lets the body make sulfamidase, the enzyme children with MPS IIIA lack. With the enzyme restored, heparan sulfate can be broken down properly instead of accumulating in the brain and body. The FDA has granted FAYUVI <strong>orphan drug, fast track, and breakthrough therapy designations<\/strong>.<\/p>\n\n\n\n<p>Sanfilippo syndrome type A is an ultra-rare, fatal lysosomal storage disease. Children typically develop normally at first, then progressively lose cognitive, language, and motor abilities. Ultragenyx estimates about <strong>3,000 to 5,000 patients<\/strong> in commercially accessible geographies, with a median life expectancy of about 15 years. Until this approval, no FDA-approved <a href=\"https:\/\/www.delveinsight.com\/report-store\/sanfilippo-syndrome-type-a-mps-iiia-market\">Sanfilippo syndrome therapy<\/a> could change the course of the disease. FDA leaders called it a historic moment for families. Patient advocacy groups, including the Cure Sanfilippo Foundation and the National MPS Society, called it the result of decades of advocacy, fundraising, and perseverance.<\/p>\n\n\n\n<p>The approval of FAYUVI is supported by data from the <strong>pivotal Transpher A study<\/strong> and long-term follow-up, with available data now extending to nearly eight years. Treatment demonstrated a reduction in heparan sulfate levels in cerebrospinal fluid across all age groups. In the primary efficacy population of 17 patients, treated children achieved cognitive scores approximately 23.5 points higher on the Bayley-III cognitive scale compared with an external natural history cohort of 27 children, according to Ultragenyx, with the company reporting a statistically significant difference of p&lt;0.0001.&nbsp;<\/p>\n\n\n\n<p>Developmental outcomes also showed meaningful differences, with eight younger or earlier-stage children reaching a cognitive developmental age of 36 months, compared with none in the natural history cohort. Among the 10 older or later-stage children, each retained function in at least one of the three assessed developmental areas, contrasting with the decline typically observed in untreated children.<\/p>\n\n\n\n<p>The FDA noted that the study was open-label, single-arm, and multicenter, with treated patients evaluated against untreated historical controls. While this study design is commonly used in ultra-rare diseases, the limitations associated with historical comparisons underscore the importance of continued long-term follow-up and post-marketing data to further characterize FAYUVI&#8217;s durability and safety profile. As with other <a href=\"https:\/\/www.delveinsight.com\/report-store\/adeno-associated-virus-vectors-in-gene-therapy-market\">AAV-based gene therapies<\/a>, FAYUVI carries several important warnings and precautions. Hepatotoxicity was the most common adverse reaction, with elevated liver enzymes reported in 85% of patients, requiring liver function monitoring and corticosteroid treatment before and after infusion. Platelet counts must also be monitored because of the risk of thrombocytopenia, with weekly monitoring recommended for the first four weeks followed by monthly assessments for six months.&nbsp;<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"436\" src=\"https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/FAYUVI-Approval-Journey-1024x436.webp\" alt=\"FAYUVI-Approval-Journey\" class=\"wp-image-36198\" srcset=\"https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/FAYUVI-Approval-Journey-1024x436.webp 1024w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/FAYUVI-Approval-Journey-300x128.webp 300w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/FAYUVI-Approval-Journey-150x64.webp 150w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/FAYUVI-Approval-Journey-768x327.webp 768w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/FAYUVI-Approval-Journey-1536x654.webp 1536w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/FAYUVI-Approval-Journey.webp 1920w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/figure>\n\n\n\n<p>Although no cases of thrombotic microangiopathy (TMA) were reported in clinical studies, the FDA includes TMA as an important warning associated with AAV therapies. Patients should also be monitored during and after administration for hypersensitivity and infusion-related reactions. In addition, FAYUVI carries a theoretical long-term malignancy risk because AAV vector DNA may integrate into the genome. Other commonly reported adverse reactions included vomiting in 67% of patients, abnormal behavior in 56%, diarrhea in 48%, and fever in 41%.<\/p>\n\n\n\n<p>Corticosteroid treatment begins one day before FAYUVI infusion and continues for at least eight weeks. Precautions related to vector shedding are recommended for three months following treatment, while vaccinations should be avoided during the 30 days preceding administration. FAYUVI must be administered in a healthcare setting equipped to monitor patients and manage potential infusion-related reactions.<\/p>\n\n\n\n<p>Ultragenyx set a wholesale acquisition cost of <strong>$3.95 million<\/strong>, a list price before discounts, rebates, or outcomes-based arrangements. This puts FAYUVI just behind <strong>Orchard Therapeutics&#8217; LENMELDY<\/strong>, priced at <strong>$4.25 million<\/strong>, among the costliest gene therapies ever launched. Ultragenyx&#8217;s other new <a href=\"https:\/\/www.delveinsight.com\/report-store\/glycogen-storage-disease-market\">glycogen storage disease gene therapy<\/a>, <strong>GENGLYCOS<\/strong>, is expected to cost <strong>$2.7 million<\/strong>. Access will run through a network of Qualified Treatment Centers in the U.S. Commercial product is expected to ship within 30 to 60 days. The company&#8217;s UltraCare program includes gene therapy guides to help families with insurance coverage and treatment logistics.<\/p>\n\n\n\n<p>FAYUVI\u2019s path to approval was far from smooth. The gene therapy\u2019s origins trace back to Nationwide Children\u2019s Hospital, where the vector was initially developed and later licensed to <strong>Abeona Therapeutics<\/strong> as <strong>ABO-102<\/strong>. In 2022, Ultragenyx secured exclusive rights to the program, with Abeona remaining eligible to receive up to <strong>$30 million<\/strong> in commercial milestones, along with tiered royalties. The road to approval faced a <strong>major setback in July 2025<\/strong>, when the FDA rejected Ultragenyx\u2019s application citing manufacturing issues at both the company\u2019s own facility and a third-party manufacturing site. However, the company resubmitted the application, and in April 2026, the FDA accepted it for priority review. That process culminated in approval on September 17, 2026.<\/p>\n\n\n\n<p>FAYUVI is manufactured in the U.S., with production taking place at Ultragenyx\u2019s facility in Bedford, Massachusetts, and at Andelyn Biosciences in Columbus, Ohio. The approval marks Ultragenyx\u2019s second gene therapy approval in less than a month, following Genglycos for glycogen storage disease type Ia, and represents the company\u2019s sixth FDA approval overall.<\/p>\n\n\n\n<p>The FAYUVI approval also delivers Ultragenyx a second priority review voucher. The company plans to monetize its vouchers. Ultragenyx had <strong>$436 million<\/strong> in cash and securities as of June 30, while its shares climbed <strong>13% to $14.50<\/strong> on the day of the approval. The milestone comes after a difficult period for the company. Ultragenyx\u2019s Angelman syndrome candidate, <strong>apazunersen<\/strong>, failed in Phase 3, triggering a more than 43% decline in the company\u2019s stock at the time and prompting \u201csignificant expense reductions.\u201d<\/p>\n\n\n\n<p>The timing of FAYUVI\u2019s approval is also notable against a broader debate over the FDA\u2019s approach to rare disease therapies. Earlier this year, rare disease advocates protested at the agency\u2019s White Oak campus over what they viewed as an overly stringent approach to rare disease approvals. Since then, the FDA approved <a href=\"https:\/\/www.delveinsight.com\/blog\/avlayah-for-hunter-syndrome\"><strong>Denali\u2019s AVLAYAH<\/strong> for Hunter syndrome (MPS II)<\/a> and withdrew a request for a control arm for Regenxbio\u2019s MPS II candidate, although Regenxbio\u2019s subsequent refiling has been delayed by a clinical hold. Against this backdrop, Ultragenyx said it hopes FAYUVI\u2019s approval will help reinvigorate investment in other ultra-rare gene therapies.<\/p>\n\n\n\n<figure class=\"wp-block-image size-large\"><a href=\"https:\/\/www.delveinsight.com\/report-store\/sanfilippo-syndrome-type-a-mps-iiia-market\"><img loading=\"lazy\" decoding=\"async\" width=\"1024\" height=\"194\" src=\"https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/Sanfilippo-Syndrome-Type-A-Market-Outlook-1024x194.webp\" alt=\"Sanfilippo Syndrome Type A Market Outlook\" class=\"wp-image-36197\" srcset=\"https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/Sanfilippo-Syndrome-Type-A-Market-Outlook-1024x194.webp 1024w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/Sanfilippo-Syndrome-Type-A-Market-Outlook-300x57.webp 300w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/Sanfilippo-Syndrome-Type-A-Market-Outlook-150x28.webp 150w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/Sanfilippo-Syndrome-Type-A-Market-Outlook-768x145.webp 768w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/Sanfilippo-Syndrome-Type-A-Market-Outlook-1536x291.webp 1536w, https:\/\/www.delveinsight.com\/blog\/wp-content\/uploads\/2026\/09\/Sanfilippo-Syndrome-Type-A-Market-Outlook.webp 1584w\" sizes=\"auto, (max-width: 1024px) 100vw, 1024px\" \/><\/a><\/figure>\n","protected":false},"excerpt":{"rendered":"<p>Summary For decades, a Sanfilippo syndrome type A diagnosis meant families could only manage symptoms. On September 17, 2026, that changed. The U.S. FDA approved Ultragenyx&#8217;s FAYUVI (rebisufligene etisparvovec-hopf), the first treatment for children with MPS IIIA that targets the underlying disease. The approval comes after a 2025 rejection, a record price tag, and a [&hellip;]<\/p>\n","protected":false},"author":14,"featured_media":36195,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":0,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","advgb_blocks_editor_width":"","advgb_blocks_columns_visual_guide":"","footnotes":""},"categories":[17],"tags":[16988,23123,258,23127,23124,6763,23126,23125],"industry":[17225],"therapeutic_areas":[17234],"class_list":["post-36193","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-articles","tag-aav-gene-therapies","tag-fayuvi","tag-gene-therapy","tag-lysosomal-storage-disease","tag-mps-iii","tag-sanfilippo-syndrome","tag-sanfilippo-syndrome-therapy","tag-sanfilippo-syndrome-type-a","industry-pharmaceutical","therapeutic_areas-rare-diseases"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO Premium plugin v27.9 (Yoast SEO v27.9) - https:\/\/yoast.com\/product\/yoast-seo-premium-wordpress\/ -->\n<title>Ultragenyx\u2019s FAYUVI Turnaround: From FDA Setback to Approval<\/title>\n<meta name=\"description\" content=\"On September 17, 2026, FDA approved FAYUVI as first treatment targeting underlying cause of Sanfilippo syndrome type A in pediatric patients\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.delveinsight.com\/blog\/fayuvi-approval-for-sanfilippo-syndrome\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Ultragenyx\u2019s FAYUVI Turnaround: From FDA Setback to Approval\" \/>\n<meta property=\"og:description\" content=\"On September 17, 2026, FDA approved FAYUVI as first treatment targeting underlying cause of Sanfilippo syndrome type A in 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href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">MPS III<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Sanfilippo syndrome<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Sanfilippo syndrome therapy<\/a>","<a href=\"https:\/\/www.delveinsight.com\/blog\/articles\/\" class=\"advgb-post-tax-term\">Sanfilippo syndrome type A<\/a>"],"unlinked":["<span class=\"advgb-post-tax-term\">AAV Gene therapies<\/span>","<span class=\"advgb-post-tax-term\">FAYUVI<\/span>","<span class=\"advgb-post-tax-term\">Gene therapy<\/span>","<span class=\"advgb-post-tax-term\">lysosomal storage disease<\/span>","<span class=\"advgb-post-tax-term\">MPS III<\/span>","<span class=\"advgb-post-tax-term\">Sanfilippo syndrome<\/span>","<span class=\"advgb-post-tax-term\">Sanfilippo syndrome therapy<\/span>","<span class=\"advgb-post-tax-term\">Sanfilippo syndrome type A<\/span>"]}},"comment_count":"0","relative_dates":{"created":"Posted 3 hours ago","modified":"Updated 5 hours ago"},"absolute_dates":{"created":"Posted on Sep 25, 2026","modified":"Updated on Sep 25, 2026"},"absolute_dates_time":{"created":"Posted on Sep 25, 2026 5:34 pm","modified":"Updated on Sep 25, 2026 9:35 am"},"featured_img_caption":"fayuvi-approval-for-sanfilippo-syndrome","series_order":"","_links":{"self":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/36193","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/users\/14"}],"replies":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/comments?post=36193"}],"version-history":[{"count":2,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/36193\/revisions"}],"predecessor-version":[{"id":36199,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/posts\/36193\/revisions\/36199"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/media\/36195"}],"wp:attachment":[{"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/media?parent=36193"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/categories?post=36193"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/tags?post=36193"},{"taxonomy":"industry","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/industry?post=36193"},{"taxonomy":"therapeutic_areas","embeddable":true,"href":"https:\/\/www.delveinsight.com\/blog\/wp-json\/wp\/v2\/therapeutic_areas?post=36193"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}