LONDON– Vertex Pharmaceuticals (Nasdaq: VRTX) announced it has reached a reimbursement agreement with the Italian Medicines Agency (AIFA) to provide access to CASGEVY® (exagamglogene autotemcel), the first and only approved gene-editing therapy for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD) in Europe.
“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,” said Ludovic Fenaux, Senior Vice President, Vertex International. “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.”
Italy is home to approximately 5,000 people aged 12 and older with TDT and about 2,300 with SCD. The agreement places Italy among a growing list of countries offering reimbursement for CASGEVY, including Austria, Bahrain, Denmark, England, Saudi Arabia and the United Arab Emirates.
TDT is a serious genetic blood disorder that requires frequent transfusions and iron chelation therapy throughout life. Patients face reduced life expectancy, diminished quality of life, and significant health care burdens. In Europe, the average age of death for TDT patients is 50 to 55 years. Symptoms can include anemia-related fatigue, shortness of breath, jaundice, feeding difficulties in infants, and complications such as organ enlargement, bone deformities, and delayed puberty.
With this latest agreement, Vertex continues to expand access to CASGEVY, a one-time CRISPR/Cas9-based therapy designed to address the root cause of disease for people living with TDT and SCD.
