Italy publicly announces reimbursement agreement for using CASGEVY®, a treatment for Transfusion-Dependent Beta Thalassemia and Sickle Cell Disease, provided by Vertex.
Vertex Pharmaceuticals, a global biotechnology company based in Boston with international headquarters in London, has announced a significant milestone. The company's non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy, CASGEVY, has been approved for the treatment of transfusion-dependent beta thalassemia (TDT) and sickle cell disease (SCD) in Italy.
CASGEVY works by editing the erythroid specific enhancer region of the BCL11A gene, leading to the production of high levels of fetal hemoglobin in red blood cells. This breakthrough therapy has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.
In the European Union, CASGEVY is approved for patients 12 years of age and older with either severe SCD with recurrent VOCs or TDT, for whom hematopoietic stem cell transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.
Italy joins a number of countries that have signed reimbursement agreements for CASGEVY, including Austria, Bahrain, Denmark, England, the Kingdom of Saudi Arabia, and the United Arab Emirates. This approval marks a significant step forward in providing access to this life-changing therapy for eligible TDT and SCD patients in Italy.
Vertex Pharmaceuticals has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases such as neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, and myotonic dystrophy type 1.
TDT is a serious, life-threatening genetic disease that requires frequent blood transfusions and iron chelation therapy throughout a person's life. SCD, on the other hand, is a debilitating, progressive, life-shortening genetic disease that affects the red blood cells and causes severe pain, organ damage, and shortened life span.
Vertex Pharmaceuticals has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia, and acute pain, and it continues to advance clinical and research programs in these areas. The company's research and development sites and commercial offices are located in North America, Europe, Australia, Latin America, and the Middle East.
The company's press release contains forward-looking statements regarding expectations for CASGEVY, access to CASGEVY for eligible TDT and SCD patients in Italy, and the population of people living with TDT and SCD in Europe. However, the risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements include data from the company's development programs not supporting registration or further development of its compounds due to safety, efficacy, and other reasons.
Investors and media can contact Vertex Pharmaceuticals Incorporated via email at [email protected] or [email protected], or by phone at +44 20 3204 5275 or 617-341-6992. The source of this news can be found at https://www.businesswire.com/news/home/20250916726929/en/.
In Italy, there are approximately 5,000 people aged 12 years and older living with TDT and around 2,300 with SCD. This approval marks a significant step forward in providing these patients with a potential cure for their debilitating diseases.
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