Vertex Reaches Reimbursement Deal for Gene Therapy in Italy

LONDON – Vertex Pharmaceuticals announced on Thursday, September 18, 2025, that it has secured a reimbursement agreement with the Italian Medicines Agency (AIFA) for its CRISPR/Cas9 gene-edited therapy, CASGEVY (exagamglogene autotemcel). This deal grants eligible patients in Italy access to the one-time, potentially curative treatment for transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD).

Italy’s agreement is particularly significant as the country has the largest TDT patient population in Europe, underscoring the urgent need for new therapeutic options.

“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 of Vertex International. “We appreciate the collaboration with AIFA to recognize the value a one-time transformative treatment provides to patients, families and the health care system.”

A Critical Milestone for Italian Patients

The reimbursement agreement opens access to CASGEVY for approximately 5,000 people aged 12 and older living with TDT and around 2,300 people with SCD in Italy. These are chronic, debilitating genetic diseases that require lifelong, resource-intensive care and significantly reduce quality of life and life expectancy.

Italy now joins a growing list of countries that have reached reimbursement deals for CASGEVY, including the United Kingdom (England), Austria, Denmark, Bahrain, the Kingdom of Saudi Arabia, and the United Arab Emirates.

How CASGEVY Works

CASGEVY is an innovative non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy. The treatment involves modifying a patient’s own hematopoietic stem and progenitor cells. Specifically, the therapy makes a precise edit at the erythroid-specific enhancer region of the BCL11A gene.

This genetic edit allows the cells to produce high levels of fetal hemoglobin (HbF), which is typically switched off after birth. By promoting HbF production, CASGEVY has been shown to reduce or eliminate debilitating vaso-occlusive crises (VOCs) in SCD patients and eliminate the need for frequent blood transfusions in TDT patients.

CASGEVY is approved in the European Union and other jurisdictions for eligible TDT and severe SCD patients aged 12 years and older who do not have a suitable human leukocyte antigen-matched related stem cell donor.