May 6, 2026 — Leads & Copy — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) has secured a reimbursement agreement with the GKV-Spitzenverband for its CRISPR/Cas9 gene-edited therapy, CASGEVY® (exagamglogene autotemcel), ensuring long-term access for eligible patients in Germany.
The agreement provides sustainable access to CASGEVY®, a one-time treatment, for individuals aged 12 years and older who are living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT).
According to Ludovic Fenaux, Senior Vice President, Vertex International, this agreement marks the first instance of a long-term, sustainable access agreement for a gene therapy in Germany for individuals with SCD and TDT.
Fenaux added that the agreement signifies significant progress for those affected by these life-shortening diseases, and Vertex is pleased to collaborate across the healthcare system to ensure the value of CASGEVY is recognized and sustainable patient access is secured.
Germany now joins a growing list of countries that have reimbursed CASGEVY, including Austria, Denmark, Italy, the Kingdom of Saudi Arabia, the United Arab Emirates, the United Kingdom, and the United States.
Vertex is committed to collaborating with government and reimbursement authorities to secure sustainable access for eligible patients worldwide.
SCD is a debilitating genetic disease affecting red blood cells, leading to severe pain, organ damage, and a shortened lifespan. Vaso-occlusive crises (VOCs), caused by blockages of blood vessels by sickled red blood cells, are a clinical hallmark of SCD.
TDT is a life-threatening genetic disease requiring frequent blood transfusions and iron chelation therapy. Complications of TDT can include an enlarged spleen, liver, or heart, misshapen bones, and delayed puberty.
CASGEVY® is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy designed for eligible patients with SCD or TDT. It involves editing a patient’s own hematopoietic stem and progenitor cells, resulting in the production of high levels of fetal hemoglobin (HbF) in red blood cells.
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. In the European Union, CASGEVY is approved for patients 12 years of age and older with either severe SCD with recurrent VOCs or TDT, for whom hematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen matched related HSC donor is not available.
Vertex is a global biotechnology company focused on creating transformative medicines for serious diseases. It has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain.
Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East.
For complete product information, please see the Summary of Product Characteristics (SmPC) at www.ema.europa.eu.
Please see full Prescribing Information including Patient Information for CASGEVY.
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Source: Vertex
