Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Sickle Cell Disease in England
Vertex Pharmaceuticals (VRTX) has secured a reimbursement agreement with NHS England for CASGEVY®, its CRISPR/Cas9 gene-edited therapy, for eligible sickle cell disease (SCD) patients. This follows the National Institute for Health and Care Excellence (NICE) issuing positive guidance recommending CASGEVY's use in the NHS.
The agreement expands upon a previous reimbursement deal reached in August 2024 for transfusion-dependent beta thalassemia (TDT) patients. Vertex is working to establish a network of independently operated authorized treatment centers (ATCs) throughout England, as the therapy requires specialized experience in stem cell transplantation and hemoglobinopathy management.
Vertex Pharmaceuticals (VRTX) ha raggiunto un accordo di rimborso con il NHS England per CASGEVY®, la sua terapia edita tramite CRISPR/Cas9, destinata ai pazienti idonei con malattia delle cellule falciformi (SCD). Questo accordo segue la pubblicazione di linee guida positive da parte del National Institute for Health and Care Excellence (NICE) che raccomandano l'uso di CASGEVY nel NHS.
L'accordo amplia un precedente contratto di rimborso raggiunto nell'agosto 2024 per i pazienti con talassemia beta dipendente da trasfusioni (TDT). Vertex sta lavorando per stabilire una rete di centri di trattamento autorizzati (ATC) autonomamente gestiti in tutta l'Inghilterra, poiché la terapia richiede esperienza specializzata nella trapianto di cellule staminali e nella gestione delle emoglobinopatie.
Vertex Pharmaceuticals (VRTX) ha firmado un acuerdo de reembolso con NHS England para CASGEVY®, su terapia editada con CRISPR/Cas9, para pacientes elegibles con enfermedad de células falciformes (SCD). Esto sigue a la emisión de orientaciones positivas por parte del National Institute for Health and Care Excellence (NICE) que recomiendan el uso de CASGEVY en el NHS.
El acuerdo expande un pacto de reembolso previo alcanzado en agosto de 2024 para pacientes con talasemia beta dependiente de transfusiones (TDT). Vertex está trabajando para establecer una red de centros de tratamiento autorizados (ATC) que operen de manera independiente en toda Inglaterra, ya que la terapia requiere experiencia especializada en trasplante de células madre y manejo de hemoglobinopatías.
Vertex Pharmaceuticals (VRTX)는 SCD(겸상 적혈구 질환) 환자를 위한 CASGEVY®, CRISPR/Cas9 유전자 편집 치료에 대해 NHS England와 환급 계약을 체결했습니다. 이는 National Institute for Health and Care Excellence (NICE)에서 CASGEVY의 NHS 사용을 권장하는 긍정적인 지침을 발표한 것에 따른 것입니다.
이번 계약은 2024년 8월에 체결된 수혈 의존성 베타 지중해빈혈(TDT) 환자를 위한 이전 환급 계약을 확장하는 것입니다. Vertex는 영국 전역에 독립적으로 운영되는 승인된 치료 센터(ATC) 네트워크를 구축하려고 노력하고 있으며, 이 치료에는 줄기 세포 이식과 헤모글로비노병 관리에 대한 전문적인 경험이 필요합니다.
Vertex Pharmaceuticals (VRTX) a obtenu un accord de remboursement avec le NHS England pour CASGEVY®, sa thérapie éditée par CRISPR/Cas9, destinée aux patients éligibles atteints de la maladie des cellules falciformes (SCD). Cela fait suite à l'émission de lignes directrices positives par le National Institute for Health and Care Excellence (NICE) recommandant l'utilisation de CASGEVY dans le NHS.
L'accord développe un ancien contrat de remboursement conclu en août 2024 pour les patients atteints de thalassémie bêta dépendante des transfusions (TDT). Vertex s'efforce d'établir un réseau de centres de traitement autorisés (ATC) opérant de manière indépendante à travers l'Angleterre, car la thérapie nécessite une expérience spécialisée dans la transplantation de cellules souches et la gestion des hémoglobinopathies.
Vertex Pharmaceuticals (VRTX) hat eine Erstattungsvereinbarung mit dem NHS England für CASGEVY®, seine CRISPR/Cas9-gene-editierte Therapie, für geeignete Patienten mit Sichelzellanämie (SCD) gesichert. Dies folgt auf die positive Empfehlung des National Institute for Health and Care Excellence (NICE), die die Verwendung von CASGEVY im NHS empfiehlt.
Die Vereinbarung erweitert einen zuvor im August 2024 erzielten Erstattungsvertrag für transfusionsabhängige beta-Thalassämie (TDT)-Patienten. Vertex arbeitet daran, ein Netzwerk von unabhängig betriebenen autorisierten Behandlungszentren (ATC) in ganz England zu schaffen, da die Therapie spezielle Erfahrungen in der Stammzelltransplantation und im Management von Hämoglobinopathien erfordert.
- Secured NHS England reimbursement agreement for CASGEVY in SCD patients
- Received positive NICE recommendation for NHS use
- Expanded market access following previous TDT reimbursement agreement
- None.
Insights
This reimbursement agreement marks a pivotal commercial achievement for Vertex's CASGEVY therapy in England. The deal's significance extends beyond immediate revenue potential, as NICE recommendations often influence other countries' reimbursement decisions, potentially accelerating global market access.
The strategic rollout through authorized treatment centers (ATCs) reflects a carefully planned commercialization strategy. While this controlled expansion ensures quality treatment delivery, it also helps manage the substantial upfront costs associated with establishing gene therapy infrastructure. This approach could serve as a blueprint for other markets, potentially smoothing the path for broader international adoption.
From a market perspective, this agreement positions Vertex advantageously in the $2+ billion global sickle cell disease market. Having secured coverage for both SCD and beta thalassemia indications in England demonstrates strong value proposition recognition by health technology assessment bodies. This dual-indication strategy effectively amortizes the infrastructure investments across a broader patient base, potentially improving the therapy's economic viability for treatment centers.
The establishment of a network of ATCs, while initially rate-limiting, creates a valuable competitive moat. These centers represent not just treatment facilities but strategic assets that could support future gene therapy launches, giving Vertex a first-mover advantage in building out the necessary ecosystem for advanced therapy delivery.
The reimbursement agreement for CASGEVY represents a paradigm shift in SCD treatment. Unlike traditional therapies that only manage symptoms, this CRISPR-based approach offers the potential for durable therapeutic benefits by addressing the genetic root cause of the disease. The expansion to cover both SCD and beta thalassemia indications demonstrates the versatility and robustness of the underlying technology platform.
The requirement for specialized stem cell transplantation expertise highlights the complexity of gene therapy administration. However, this centralized treatment model through ATCs ensures optimal patient outcomes while facilitating long-term follow-up essential for novel genetic therapies. The careful selection and preparation of treatment centers will be important for maintaining high success rates and gathering real-world evidence.
From a healthcare economics perspective, while the upfront costs are substantial, the long-term value proposition is compelling. By potentially reducing or eliminating the need for chronic care, regular transfusions and hospitalizations, CASGEVY could significantly decrease the lifetime healthcare burden for these patients. This aligns with the NHS's focus on value-based healthcare and could set precedents for future advanced therapy pricing models.
- Agreement means eligible sickle cell disease (SCD) patients in
- Agreement for CASGEVY in transfusion-dependent beta thalassemia (TDT) was previously reached in August 2024 -
The reimbursement agreement comes as the National Institute for Health and Care Excellence (NICE) issues positive guidance recommending CASGEVY’s use in the NHS. It means that eligible SCD patients in
“Today is an important day for the sickle cell community who have gone too long without treatments that address the underlying cause of their devastating disease,” said Ludovic Fenaux, Senior Vice President, Vertex International. “We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with CASGEVY, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system.”
The administration of the therapy requires experience in stem cell transplantation and the management of hemoglobinopathies; therefore, Vertex is continuing to engage with experienced hospitals throughout
About Sickle Cell Disease (SCD)
SCD is a debilitating, progressive, life-shortening genetic disease. SCD patients report health-related quality of life scores well below the general population and significant health care resource utilization. SCD affects the red blood cells, which are essential for carrying oxygen to all organs and tissues of the body. SCD causes severe pain, organ damage and shortened life span due to misshapen or “sickled” red blood cells. The clinical hallmark of SCD is vaso-occlusive crises (VOCs), which are caused by blockages of blood vessels by sickled red blood cells and result in severe and debilitating pain that can happen anywhere in the body at any time. SCD requires lifelong treatment and significant use of health care resources, and ultimately results in reduced life expectancy, decreased quality of life and reduced lifetime earnings and productivity. In
About CASGEVY® (exagamglogene autotemcel)
CASGEVY® is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.
CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world. The Conditional Marketing Authorization in
For full details about access eligibility please refer to the NICE final draft guidance issued today.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple serious diseases and conditions — cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain — and continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.
Vertex was founded in 1989 and has its global headquarters in
(VRTX-GEN)
Vertex Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the statements by Ludovic Fenaux, in this press release, and statements regarding Vertex’s expectations for and the anticipated benefits of CASGEVY, expectations for access to CASGEVY for eligible SCD patients in
View source version on businesswire.com: https://www.businesswire.com/news/home/20250129242714/en/
Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com
Media:
mediainfo@vrtx.com
or
International: +44 20 3204 5275
Source: Vertex Pharmaceuticals
FAQ
What is the new reimbursement agreement for CASGEVY (VRTX) in England?
When did VRTX secure the TDT reimbursement agreement for CASGEVY in England?
How will CASGEVY be administered to patients in England?
What regulatory body approved CASGEVY for NHS use in England?
