Improving DxMultiomics, Powered by Your Choices

We're building DxMultiomics to be The Central Nexus of Biotech Intelligence. To ensure a seamless experience and continuous improvement, we utilize website analytics. These tools help us monitor site performance, understand how users navigate the platform, and identify opportunities to make your research journey more efficient. We handle this data with the utmost respect for your privacy, and you can choose what works best for you.

Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Transfusion-Dependent Beta Thalassemia and Sickle Cell Disease
_↗ investors.vrtx.com
News Image

An agreement has been reached between the Italian Medicines Agency (AIFA) and Vertex Pharmaceuticalsfor the reimbursement of CASGEVY® (exagamglogene autotemcel), a gene-editing therapy intended for eligible patients with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). This agreement is particularly significant for Italy, as the country has the largest population of people living with TDT in Europe, with approximately 5,000 people aged 12 and older affected by the disease. An additional 2,300 people in Italy live with SCD. This milestone makes CASGEVY the first and only gene-editing therapy to be reimbursed for TDT and SCD in Europe, joining other countries such as Austria, Bahrain, Denmark, England, the Kingdom of Saudi Arabia, and the United Arab Emirates. for the reimbursement of CASGEVY® (exagamglogene autotemcel), a gene-editing therapy intended for eligible patients with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). This agreement is particularly significant for Italy, as the country has the largest population of people living with TDT in Europe, with approximately 5,000 people aged 12 and older affected by the disease. An additional 2,300 people in Italy live with SCD. This milestone makes CASGEVY the first and only gene-editing therapy to be reimbursed for TDT and SCD in Europe, joining other countries such as Austria, Bahrain, Denmark, England, the Kingdom of Saudi Arabia, and the United Arab Emirates.

Both TDT and SCD are serious, life-threatening genetic diseases that require lifelong treatment and have a significant impact on a patient's quality of life. TDT is characterized by a need for frequent blood transfusions and iron chelation therapy, while SCD is marked by severe pain crises (vaso-occlusive crises) and organ damage from misshapen red blood cells. The mean age of death in Europe for TDT patients is 50-55 years, and for SCD patients, it is around 40 years. CASGEVY is a one-time, non-viral, gene-edited cell therapy that modifies a patient's own stem cells to produce high levels of fetal hemoglobin. This has been shown to reduce or eliminate the need for transfusions in TDT patients and vaso-occlusive crises in SCD patients. The approval of CASGEVY in Europe is for patients 12 years and older with severe SCD or TDT who are candidates for stem cell transplantation but do not have a matched related donor. This reimbursement agreement with Italy's AIFA represents a turning point for patients, offering a transformative, one-time treatment that addresses a significant unmet medical need and provides a valuable alternative to the lifelong management of these chronic conditions.

Log In
Sign Up