Vertex Announces Reimbursement Agreement with NHS England for CASGEVY™
Vertex has announced a new reimbursement agreement with NHS England, effective immediately, allowing eligible patients with transfusion-dependent beta thalassemia (TDT) to access CASGEVY™ (exagamglogene autotemcel), a CRISPR/Cas9 gene-edited therapy. This agreement follows positive guidance from the National Institute for Health and Care Excellence (NICE) recommending CASGEVY’s use within the NHS.
CASGEVY received the world’s first authorization for a CRISPR-based gene-editing therapy from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on November 15, 2023.
Ludovic Fenaux, Senior Vice President at Vertex International, remarked, “This agreement marks a historic moment for those with transfusion-dependent beta thalassemia, a condition with historically limited treatment options. Our collaboration with NHS England and NICE underscores the significant value of this one-time treatment for patients, families, and the healthcare system.”
Administering CASGEVY requires expertise in stem cell transplantation and the management of hemoglobinopathies. Vertex is working with experienced hospitals across England to establish authorized treatment centers (ATCs).
Vertex is also partnering with NICE and NHS England to facilitate access to CASGEVY for sickle cell disease (SCD) patients in England and is engaging with European authorities to expand access to this innovative therapy for both SCD and TDT patients.
About Transfusion-Dependent Beta Thalassemia (TDT)
TDT is a severe genetic disorder requiring lifelong blood transfusions and iron chelation therapy. Patients with TDT often face reduced life expectancy and significant healthcare needs. Complications can include anemia, fatigue, organ enlargement, and delayed puberty. The mean age of death for TDT patients in Europe is 50-55 years, and stem cell transplantation is a potential but limited curative option due to donor availability.
CASGEVY™ is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy. It involves editing a patient’s own hematopoietic stem and progenitor cells to produce high levels of fetal hemoglobin, which can reduce or eliminate symptoms in both SCD and TDT patients. It has been approved for certain indications in multiple regions, including Great Britain, where it has Conditional Marketing Authorization for TDT and SCD patients with specific genotypes.
Vertex is a global biotechnology company focused on developing transformative medicines for serious diseases. Its approved therapies target the underlying causes of cystic fibrosis, sickle cell disease, and transfusion-dependent beta thalassemia. Vertex also has a robust pipeline of investigational therapies for other serious conditions.
Founded in 1989, Vertex is headquartered in Boston with international offices in London and other global locations. The company is frequently recognized as a top employer and continues to innovate in the field of biotechnology.
