A new era in gene therapy has been officially ushered in with the European Commission’s conditional marketing authorization of Vertex Pharmaceuticals Incorporated’s CASGEVY™ (exagamglogene autotemcel [exa-cel]). This transformative CRISPR/Cas9 gene-editing therapy is now authorized for the treatment of patients 12 years of age and older with transfusion-dependent beta-thalassemia (TDT) or severe sickle cell disease (SCD), which is characterized by recurrent vaso-occlusive crises. These patients are candidates for hematopoietic stem cell transplantation but do not have a compatible related donor who matches their antigen.
What is CASGEVY™?
CASGEVY™, the only genetic therapy approved for individuals with SCD and TDT in the European Union, is a significant development, with over 8,000 patients now eligible for this innovative treatment. According to Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex, “This approval not only represents an important milestone in the treatment of these serious genetic disorders, but it also increases the potential to significantly improve the lives of tens of thousands of patients worldwide.”
The devastating and life-limiting nature of sickle cell disease and transfusion-dependent beta thalassemia places a significant strain on patients, families, and healthcare systems. CASGEVY™ is a significant advancement, promising a functional treatment. As noted by Franco Locatelli, M.D., Ph.D., the principle investigator in key studies and a major figure in pediatric hematology, the availability of this therapeutic alternative for eligible patients is critical.
Vertex is working closely with national health authorities to improve access for eligible patients, and has already gained early access for eligible TDT patients in France. CASGEVY™ will be administered by around 25 independently operated authorized treatment centers (ATCs) across Europe.
Sickle Cell Disease (SCD)
Sickle cell disease and transfusion-dependent beta-thalassemia are characterized by significant health challenges and impaired quality of life, requiring lifelong treatment and extensive healthcare resources. The introduction of CASGEVY™, a non-viral ex vivo CRISPR/Cas9 gene-edited cell therapy, represents a significant advance in the treatment of these diseases. It offers the potential to reduce or eliminate disease symptoms by enhancing fetal hemoglobin production in red blood cells.
Vertex Pharmaceuticals, a worldwide biotechnology company, continues to drive scientific innovation by developing transformational therapies for critical diseases. This approval solidifies Vertex’s position at the forefront of biotechnological developments by highlighting its dedication to enhancing patient outcomes and quality of life for people with chronic, life-limiting genetic illnesses.
Source: Vertex News & Events
