{"id":82377,"date":"2026-07-02T00:05:36","date_gmt":"2026-07-02T00:05:36","guid":{"rendered":"https:\/\/english.saigonbiz.com\/vn\/fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease\/"},"modified":"2026-07-02T00:05:36","modified_gmt":"2026-07-02T00:05:36","slug":"fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease","status":"publish","type":"post","link":"https:\/\/english.saigonbiz.com\/vn\/fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease\/","title":{"rendered":"FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease"},"content":{"rendered":"
\n

State-of-the-art gene therapy provides new treatment option for children 2 years and older with life-threatening blood disorder<\/em><\/p>\n

Silver Spring, MD, July 01, 2026 (GLOBE NEWSWIRE) — The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent \u03b2 thalassemia (TDT). This is the first gene therapy approved for patients aged 2 years and older with SCD.<\/p>\n

Casgevy has been previously approved for the treatment of patients aged 12 years and older with SCD with recurrent VOCs or TDT.<\/p>\n

\u201cWith today\u2019s decision, pediatric patients as young as 2 years of age can now access a critical additional treatment option to treat these debilitating, life-threatening diseases,\u201d said Karim Mikhail, B. Pharm., M.S., Acting Director of the Center for Biologics Evaluation and Research (CBER). \u201cThe FDA is committed to prioritizing and speeding up the review of products that address critical U.S health priorities through expedited review programs, including the FDA Commissioner\u2019s National Priority Voucher (CNPV) Pilot Program. These initiatives are designed to advance therapies for diseases with significant unmet medical needs, enabling faster access to innovative treatments while upholding the FDA\u2019s rigorous gold-standard requirements for safety and effectiveness.\u201d<\/p>\n

SCD affects red blood cells (RBCs), which contain hemoglobin, a protein that transports oxygen throughout the body. SCD can cause various symptoms and health issues, including episodes of severe pain called sickle cell crises or VOCs.<\/p>\n

Thalassemia is a genetic blood disorder that causes the body to have an abnormally low level of hemoglobin, resulting in reduced oxygen delivery to the body\u2019s tissues. In some cases, individuals require regular blood transfusions to maintain adequate levels of functional hemoglobin.<\/p>\n

Casgevy is a gene therapy consisting of the patient\u2019s own (autologous) hematopoietic (blood) stem cells, administered as a one-time single dose for intravenous infusion. The cells are edited using CRISPR\/Cas9, a type of genome editing technology, and then engrafted in the body\u2019s bone marrow. CRISPR\/Cas9 can be directed to a specific spot in DNA, where it cuts the genetic material so that DNA can be accurately removed, added, or replaced. In patients with severe SCD, this treatment increases a type of hemoglobin which is called fetal hemoglobin (HbF). This helps prevent RBCs from forming into abnormal sickle shapes and addresses the underlying cause of disease, thereby eliminating VOCs.<\/p>\n

In patients with TDT, treatment increases HbF levels and total hemoglobin levels, hence eliminating dependence on regular RBC transfusions.<\/p>\n

Full myeloablative conditioning, a high-intensity preparatory treatment given to patients before they receive a stem cell transplant or gene therapy, is administered prior to treatment with Casgevy.<\/p>\n

\u201cThese disorders carry a heavy burden for children and their families, affecting growth, development, and long-term health in profound ways,\u201d\u00a0said Megha Kaushal M.D., MSc, Acting Deputy Director of the Office of Therapeutic Products in CBER and pediatric hematologist. \u201cGrounded in the scientific evidence that earlier treatment reduces the risk of lasting end-organ damage, making this therapy available to younger patients opens a critical window for intervention and gives these children a meaningful chance at a healthier future.\u201d<\/p>\n

The safety and effectiveness of Casgevy in patients aged 5 years to less than 12 years with SCD were evaluated in a clinical trial which included 11 patients. All eight patients who were evaluable for efficacy achieved the primary efficacy outcome of VF12 (no protocol-defined severe VOCs for at least 12 consecutive months within the first 24 months after infusion with Casgevy).<\/p>\n

The efficacy and safety of Casgevy in patients 5 years to less than 12 years of age with TDT were evaluated in a trial of 15 patients. Eight of the nine efficacy evaluable patients with TDT achieved transfusion independence for 12 consecutive months, with a median duration of transfusion independence of 20.1 months.<\/p>\n

Based on product characteristics and clinical study data, extrapolation to the younger pediatric age population was granted to expand the indication to 2 years of age and above for both conditions.<\/p>\n

The most common adverse reactions were mucositis (inflammation of mucous membranes) and febrile neutropenia (fever associated with low levels of a type of white blood cell called neutrophils) in patients with SCD and in patients with TDT, and decreased appetite in patients with SCD. Additionally, the prescribing information contains warnings for neutrophil engraftment failure, delayed platelet engraftment, hypersensitivity reactions, and off-target genome editing risk (refers to the possibility that the CRISPR\/Cas9 editing process may make unintended edits to parts of the genome other than the intended target site).<\/p>\n

The approval decision was granted just 53 days after filing and represented the eighth approval selected for the\u00a0Commissioner’s National Priority Voucher (CNPV) pilot program<\/a>. The FDA granted Casgevy\u00a0Orphan Drug<\/a>,\u00a0regenerative medicine advanced therapy (RMAT)<\/a>, and\u00a0Fast Track<\/a> designations.<\/p>\n

The FDA granted the approval to Vertex Pharmaceuticals, Incorporated.<\/p>\n

Contact Info<\/p>\n

U.S. Food and Drug Administration
FDAPressAlerts@fda.hhs.gov<\/a>
+1 202-690-6343<\/p>\n

\"\"<\/div>\n","protected":false},"excerpt":{"rendered":"

State-of-the-art gene therapy provides new treatment option for children 2 years and older with life-threatening blood disorder Silver Spring, MD, July 01, 2026 (GLOBE NEWSWIRE) — The U.S. Food and Drug Administration today issued a supplemental approval for Casgevy (exagamglogene autotemcel) for patients aged 2 years and older with either sickle cell disease (SCD) with […]\n","protected":false},"author":1,"featured_media":85254,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[5],"tags":[],"class_list":["post-82377","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-people"],"yoast_head":"\nFDA Approves First Gene Therapy for Young Children with Sickle Cell Disease - Biz and Live in Saigon<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/english.saigonbiz.com\/vn\/fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease\" \/>\n<meta property=\"og:description\" content=\"State-of-the-art gene therapy provides new treatment option for children 2 years and older with life-threatening blood disorder Silver Spring, MD, Jul\" \/>\n<meta property=\"og:url\" content=\"https:\/\/english.saigonbiz.com\/vn\/fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease\/\" \/>\n<meta property=\"og:site_name\" content=\"Biz and Live in Saigon\" \/>\n<meta property=\"article:publisher\" content=\"https:\/\/www.facebook.com\/SaigonbizGroup\" \/>\n<meta property=\"article:published_time\" content=\"2026-07-02T00:05:36+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/english.saigonbiz.com\/vn\/wp-content\/uploads\/2026\/05\/SaigonBiz-Feature-Image.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"1074\" \/>\n\t<meta property=\"og:image:height\" content=\"716\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\n<meta name=\"author\" content=\"admin\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"admin\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"4 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\/\/english.saigonbiz.com\/vn\/fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease\/#article\",\"isPartOf\":{\"@id\":\"https:\/\/english.saigonbiz.com\/vn\/fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease\/\"},\"author\":{\"name\":\"admin\",\"@id\":\"https:\/\/english.saigonbiz.com\/vn\/#\/schema\/person\/267b348e6cf32a66aab33637c2d399a6\"},\"headline\":\"FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease\",\"datePublished\":\"2026-07-02T00:05:36+00:00\",\"dateModified\":\"2026-07-02T00:05:36+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\/\/english.saigonbiz.com\/vn\/fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease\/\"},\"wordCount\":822,\"publisher\":{\"@id\":\"https:\/\/english.saigonbiz.com\/vn\/#organization\"},\"articleSection\":[\"People\"],\"inLanguage\":\"en-US\"},{\"@type\":\"WebPage\",\"@id\":\"https:\/\/english.saigonbiz.com\/vn\/fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease\/\",\"url\":\"https:\/\/english.saigonbiz.com\/vn\/fda-approves-first-gene-therapy-for-young-children-with-sickle-cell-disease\/\",\"name\":\"FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease - 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