The annual American Society of Hematology (ASH) conference provided the stage for groundbreaking findings on CASGEVY® (exa-cel), the innovative therapy developed by Vertex Pharmaceuticals. These results highlight a sustained impact for patients with sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT), conditions that significantly affect quality of life.
Clinical benefits of exa-cel
With over five years of patient follow-up, the CLIMB-111 and CLIMB-121 studies revealed remarkable outcomes:
- 93% of SCD patients remained free from vaso-occlusive crises (VOC) for at least one year.
- 98% of TDT patients achieved transfusion independence for at least 12 consecutive months.
These findings underscore exa-cel’s potential to enhance not only clinical parameters but also the quality of life, with notable improvements in physical, emotional, and social well-being.
Safety and innovative technology
Exa-cel, powered by CRISPR/Cas9 technology, employs ex vivo gene editing to boost fetal hemoglobin (HbF) production, reducing or eliminating the need for transfusions and painful crises. Its safety profile aligns with expectations for autologous hematopoietic stem cell transplants.
Global access to exa-cel
Currently approved in countries such as the United States, European Union, Canada, and Saudi Arabia, exa-cel is set for further expansion. Over 45 treatment centers are operational globally, and groundbreaking agreements, like the one with the Centers for Medicare & Medicaid Services (CMS) in the US, ensure broader and equitable access.
Pioneering new frontiers in care
Vertex continues to lead in biotechnological innovation, addressing severe diseases like cystic fibrosis, type 1 diabetes, and neuropathic pain, solidifying its position as a global biotech leader.
Exa-cel stands as a milestone in medicine, offering patients a one-time treatment with lifelong benefits.
