{"id":978059,"date":"2026-07-01T20:01:08","date_gmt":"2026-07-02T00:01:08","guid":{"rendered":"https:\/\/www.marketnewsdesk.com\/index.php\/vertex-announces-us-fda-approval-for-expanded-use-of-casgevy-for-the-treatment-of-people-ages-2-years-and-older-with-sickle-cell-disease-or-transfusion-dependent-beta-thalassemia\/"},"modified":"2026-07-01T20:01:08","modified_gmt":"2026-07-02T00:01:08","slug":"vertex-announces-us-fda-approval-for-expanded-use-of-casgevy-for-the-treatment-of-people-ages-2-years-and-older-with-sickle-cell-disease-or-transfusion-dependent-beta-thalassemia","status":"publish","type":"post","link":"https:\/\/www.marketnewsdesk.com\/index.php\/vertex-announces-us-fda-approval-for-expanded-use-of-casgevy-for-the-treatment-of-people-ages-2-years-and-older-with-sickle-cell-disease-or-transfusion-dependent-beta-thalassemia\/","title":{"rendered":"Vertex Announces US FDA Approval for Expanded Use of CASGEVY\u00ae for the Treatment of People Ages 2 Years and Older With Sickle Cell Disease or Transfusion-Dependent Beta Thalassemia"},"content":{"rendered":"

<\/p>\n

Vertex Announces US FDA Approval for Expanded Use of CASGEVY\u00ae<\/sup> for the Treatment of People Ages 2 Years and Older With Sickle Cell Disease or Transfusion-Dependent Beta Thalassemia<\/b><\/p>\n

– First and only approved genetic therapy to treat children as young as 2 years for both severe sickle cell disease and transfusion-dependent beta thalassemia –<\/i><\/p>\n

– Approximately 5,500 additional children in the U.S. are now eligible for this established one-time therapy, expanding upon the prior FDA approval in people 12 years and older –<\/i><\/p>\n

– Regulatory review for label expansion underway in the Kingdom of Saudi Arabia and United Kingdom –<\/i><\/p>\n

BOSTON–(BUSINESS WIRE<\/a>)–Vertex Pharmaceuticals Incorporated<\/a> (Nasdaq: VRTX) announced today that the U.S. Food and Drug Administration (FDA) has approved expanded use of CASGEVY\u00ae<\/sup> (exagamglogene autotemcel) for the treatment of people ages 2 years and older with either sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta thalassemia (TDT). CASGEVY is the first approved genetic therapy indicated for children as young as 2 years for both SCD and TDT.<\/p>\n

\n\u201cJust as we redefined what is possible in cystic fibrosis, our ambition is to transform the future for people living with sickle cell disease and transfusion-dependent beta thalassemia. The remarkable consistency of results across age groups reinforces the potential of CASGEVY to deliver durable, transformative benefits to those who have historically had limited options,\u201d said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. \u201cWe\u2019re deeply grateful to the patients, families and investigators who participated in the clinical trials that led to this historic approval, and we are ready to bring CASGEVY to children and their families across the U.S.\u201d<\/p>\n

\n\u201cToday\u2019s approval offers renewed hope for children living with sickle cell disease or transfusion\u2011dependent beta thalassemia,\u201d said Haydar Frangoul, M.D., M.S., Medical Director of HCA Healthcare\u2019s Sarah Cannon Transplant and Cellular Therapy Program at TriStar Centennial Children\u2019s Hospital, investigator with Sarah Cannon Research Institute (SCRI) and Member of Vertex\u2019s SCD Program Steering Committee. \u201cEarlier access to the transformative potential of this therapy will allow clinicians and families to consider treatment before years of cumulative damage from these life-shortening diseases take hold.\u201d<\/p>\n

\nVertex has established a network of independently operated, authorized treatment centers (ATCs) throughout the U.S. to offer CASGEVY to eligible patients through existing access and reimbursement pathways. Today, there are more than 75 activated ATCs in the U.S. The full list can be accessed at CASGEVY.com<\/a>.<\/p>\n

About Sickle Cell Disease (SCD)<\/b><\/p>\n

\nSickle cell disease (SCD) is a rare serious, inherited blood disease that is progressive and life\u2011shortening. The disease causes red blood cells to become rigid and misshapen, restricting blood flow and oxygen delivery to vital organs. Recurrent vaso\u2011occlusive crises (VOCs), unpredictable episodes of severe pain caused by blocked blood vessels, are a defining feature of SCD and frequently require hospitalization. Many patients experience these complications early in life, and over time, repeated VOCs and chronic anemia lead to progressive and irreversible organ damage, including damage to the brain, lungs, kidneys and heart. SCD places a substantial burden on patients and their families, who must manage frequent medical visits, hospitalizations, school and work disruptions, and the emotional toll of chronic pain and life\u2011threatening complications. Despite lifelong treatment, people with SCD and recurrent VOCs in the U.S. face shortened life expectancy, with a median age of death of approximately 45 years, report quality\u2011of\u2011life scores far below the general population and the estimated lifetime healthcare costs of managing the disease are $4\u20136 million.<\/p>\n

About Transfusion\u2011Dependent Beta Thalassemia (TDT)<\/b><\/p>\n

\nTransfusion\u2011dependent beta thalassemia (TDT) is a rare serious, inherited blood disease that is progressive and life\u2011shortening. The disease impairs the body\u2019s ability to produce sufficient hemoglobin, limiting oxygen delivery to tissues and organs. People with TDT do not have enough functional hemoglobin in their red blood cells and require regular, lifelong blood transfusions, often beginning early in childhood, along with ongoing iron chelation therapy. While transfusions are necessary for survival, many of the long\u2011term complications of TDT are exacerbated by chronic transfusion therapy and iron overload and cumulative damage to the heart, liver and endocrine system, as well as bone abnormalities and delayed growth and puberty. TDT places a significant and ongoing burden on patients and their families, requiring frequent medical visits and complex lifelong treatment. Despite lifelong treatment, people with TDT in the U.S. face shortened life expectancy, with a median age of death of approximately 37 years, reduced quality of life and productivity, and the estimated lifetime healthcare costs of managing the disease are $5\u20135.7 million.<\/p>\n

About CASGEVY\u00ae<\/sup> (exagamglogene autotemcel)<\/b><\/p>\n

\nCASGEVY is a non-viral, ex vivo<\/i> CRISPR\/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient\u2019s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A<\/i> gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown in clinical trials to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.<\/p>\n

\nIn the United States, CASGEVY was approved using the Commissioner\u2019s National Priority Voucher. Vertex has also recently completed regulatory submissions in the Kingdom of Saudi Arabia and United Kingdom to expand the use of CASGEVY to children as young as five.<\/p>\n

About the CLIMB Studies<\/b><\/p>\n

\nThe completed Phase 1\/2\/3 open-label studies, CLIMB-111 and CLIMB-121, were designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12-35 years with TDT or with SCD and recurrent VOCs. Patients were followed for approximately two years after CASGEVY infusion in these studies. CLIMB-141 and CLIMB-151 are ongoing Phase 3 open-label studies, designed to assess the safety and efficacy of a single dose of exagamglogene autotemcel in patients ages 2-11 years with TDT or with SCD and recurrent VOCs. Enrollment and dosing are complete for the 5\u201311-year-old cohort in both studies.<\/p>\n

\nEach patient in these studies is asked to participate in the ongoing long-term, open-label study, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients with up to 15 years of follow up after CASGEVY infusion.<\/p>\n

U.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY<\/b><\/p>\n

INDICATION<\/b><\/p>\n

\nCASGEVY is indicated for the treatment of patients aged 2 years and older with:<\/p>\n