Vertex Secures Italian Reimbursement For CASGEVY In Beta Thalassemia And Sickle Cell Disease

Vertex Pharmaceuticals announced that it has reached a reimbursement agreement with the Italian Medicines Agency (AIFA), enabling eligible patients in Italy with transfusion-dependent beta thalassemia (TDT) or severe sickle cell disease (SCD) to access CASGEVY® (exagamglogene autotemcel), its CRISPR/Cas9-based gene-editing therapy.

In Italy, more than 5,000 people aged 12 and above live with TDT, while around 2,300 are affected by SCD. With this agreement, Italy joins a growing list of countries, including Austria, Bahrain, Denmark, England, Saudi Arabia, and the United Arab Emirates, that have secured reimbursement pathways for CASGEVY.

“Today is a turning point for eligible people in Italy living with transfusion-dependent beta thalassemia and sickle cell disease, two life-shortening diseases with limited treatment options. Italy has the largest TDT population in Europe, which underscores the importance of this agreement. We appreciate the collaboration with AIFA to recognize the value a one-time transformative treatment provides to patients, families and the health care system,” said Ludovic Fenaux, Senior Vice President, Vertex International. 

TDT is a severe, inherited blood disorder that requires frequent blood transfusions and lifelong iron chelation therapy. Patients often experience fatigue, anemia-related complications, delayed development in childhood, and organ damage, leading to reduced quality of life and shortened life expectancy. In Europe, the average life expectancy for TDT patients is approximately 50–55 years. Access to CASGEVY offers a potential one-time treatment option that targets the underlying genetic cause of the disease.