Vertex Pharmaceuticals (Nasdaq: VRTX) announced today that it has secured a reimbursement agreement with NHS England, providing eligible sickle cell disease (SCD) patients access to its groundbreaking CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autotemcel). This agreement represents a significant advancement in the treatment of SCD, a debilitating and life-shortening genetic condition that has long lacked effective treatment options addressing the disease’s underlying causes.
“We are pleased to have reached this new agreement that ensures both eligible SCD and transfusion-dependent beta thalassemia (TDT) patients can now access CASGEVY. This is a milestone that reflects the profound value that a one-time treatment like CASGEVY can provide not only to patients and their families but also to the healthcare system,” said Vertex Pharmaceuticals in a statement. The agreement also follows the earlier one made in August for transfusion-dependent beta thalassemia (TDT) patients, allowing both groups to benefit from the potential of CASGEVY.
CASGEVY’s inclusion in the NHS following a positive recommendation from the National Institute for Health and Care Excellence (NICE) highlights the growing recognition of the therapy’s potential in treating SCD. This recommendation follows several important clinical and regulatory milestones, ensuring that patients in England who meet the criteria will have access to this promising new treatment option.
The CRISPR/Cas9 gene-edited therapy works by modifying the patient’s own hematopoietic stem and progenitor cells to produce increased levels of fetal hemoglobin (HbF) in red blood cells. This change aims to reduce or eliminate the debilitating vaso-occlusive crises (VOCs), a hallmark of sickle cell disease, which can cause severe and unpredictable pain due to blockages in blood vessels. By editing the genes in these blood cells, CASGEVY offers a potential curative solution for patients who have had limited options beyond symptomatic management or, in rare cases, stem cell transplantation.
“Today marks an important day for the sickle cell community, who have gone too long without effective treatments that target the underlying cause of their devastating disease,” said Ludovic Fenaux, Senior Vice President of Vertex International. He continued, “We are delighted to announce this new agreement that ensures both eligible SCD and TDT patients can access CASGEVY. This groundbreaking one-time treatment represents a substantial advancement in how we approach genetic diseases and offers hope for patients whose lives have been overshadowed by constant medical challenges.”
The introduction of CASGEVY in England is a result of extensive collaboration between Vertex Pharmaceuticals, NICE, and NHS England. The company is continuing its efforts to expand access to this life-changing therapy by engaging with hospitals experienced in stem cell transplantation and the management of hemoglobinopathies to establish a network of independently operated authorized treatment centers (ATCs). This network will be crucial in ensuring that SCD patients can receive the therapy under the guidance of experienced medical professionals.
About Sickle Cell Disease (SCD)
Sickle Cell Disease is a severe, progressive genetic condition that primarily affects individuals of African, Mediterranean, Middle Eastern, and Indian ancestry. SCD involves the production of abnormal hemoglobin, causing red blood cells to become misshapen or “sickled.” These sickled cells can block blood flow and cause painful episodes called vaso-occlusive crises (VOCs), which result in intense and unpredictable pain, organ damage, and ultimately, a reduced life expectancy. In Europe, the mean age of death for individuals with SCD is approximately 40 years.
The disease requires ongoing management, including frequent hospital visits, blood transfusions, and pain management. While stem cell transplants from a matched donor can offer a potential cure, they are not widely accessible due to the limited number of suitable donors available, especially for people from underrepresented ethnic groups. Therefore, alternative therapies that address the root cause of SCD are desperately needed.
About CASGEVY® (exagamglogene autotemcel)
CASGEVY® is a non-viral, ex vivo CRISPR/Cas9 gene-editing therapy developed for SCD and transfusion-dependent beta thalassemia (TDT). It involves editing a patient’s own hematopoietic stem and progenitor cells, specifically targeting the erythroid-specific enhancer region of the BCL11A gene. This precise genetic modification results in the production of higher levels of fetal hemoglobin (HbF), a form of hemoglobin that is naturally present during fetal development. The production of HbF in adult patients with SCD has been shown to reduce or eliminate the painful VOCs associated with the disease, as well as the need for transfusions in patients with TDT.
Vertex Vertex
The therapy is currently approved for use in eligible patients aged 12 years and older in multiple regulatory regions globally. In Great Britain, CASGEVY has received Conditional Marketing Authorization for the treatment of SCD and TDT patients who meet specific genetic criteria and for whom a stem cell transplant from a matched donor is not available. This represents a significant breakthrough, offering a potential life-changing treatment option to those who have previously had few alternatives.
The pivotal clinical trials have demonstrated the ability of CASGEVY to significantly improve patients’ quality of life by reducing the frequency and severity of VOCs in those with SCD, and reducing the need for regular blood transfusions in TDT patients. The therapy’s promising results have been recognized by health authorities, leading to its regulatory approvals in various countries, and its positive guidance from NICE paves the way for its wider use in clinical practice.
Vertex Pharmaceuticals: Innovation in Genetic Disease Treatment
Vertex Pharmaceuticals is a leading global biotechnology company focused on scientific innovation to create transformative medicines for people with serious diseases. Vertex has a track record of developing and bringing to market approved medicines that treat the underlying causes of genetic diseases, including cystic fibrosis, sickle cell disease, and transfusion-dependent beta thalassemia.
The company’s commitment to advancing treatments for genetic diseases is evident in its ongoing research and development efforts in areas like neuropathic pain, kidney disease, and type 1 diabetes. Vertex is widely recognized for its strong pipeline of investigational therapies and has made significant strides in addressing diseases that have long been underserved by current medical treatments.
In addition to its groundbreaking work in gene therapy, Vertex has established itself as one of the best places to work in the biotechnology industry, with a reputation for fostering innovation and providing employees with an environment where they can thrive. The company was founded in 1989 and has its global headquarters in Boston, Massachusetts. Its international operations include research and development sites, as well as commercial offices in regions across North America, Europe, and Asia.
