BOSTON — Vertex Pharmaceuticals Inc. said it has signed a reimbursement agreement in Germany for CASGEVY, its CRISPR/Cas9 gene-edited cell therapy for severe sickle cell disease and transfusion-dependent beta thalassemia.
The agreement with GKV-Spitzenverband, Germany’s national association of statutory health insurance funds, secures access to the one-time treatment for eligible patients in Germany ages 12 and older.
“For the first time in Germany, a long-term, sustainable access agreement to a gene therapy has been established for people living with sickle cell disease and transfusion-dependent beta thalassemia,” said Ludovic Fenaux, Senior Vice President, Vertex International. “This agreement represents significant progress for people living with these two devastating and life-shortening diseases. We are pleased to collaborate across the health care system to ensure the value of CASGEVY is recognized and sustainable patient access is secured.”
Germany joins several other countries that have reimbursed CASGEVY, including Austria, Denmark, Italy, Saudi Arabia, the United Arab Emirates, the United Kingdom and the United States. Vertex said it continues to work with government and reimbursement authorities globally to expand access for eligible patients.
CASGEVY, also known as exagamglogene autotemcel, is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy. The treatment uses a patient’s own hematopoietic stem and progenitor cells, which are edited at the erythroid-specific enhancer region of the BCL11A gene. The edit is intended to increase production of fetal hemoglobin in red blood cells.
Vertex said CASGEVY has been shown to reduce or eliminate vaso-occlusive crises in patients with sickle cell disease and reduce or eliminate transfusion requirements in patients with transfusion-dependent beta thalassemia.
Sickle cell disease is a genetic disorder that affects red blood cells and can cause severe pain, organ damage and shortened life expectancy. Vaso-occlusive crises, a hallmark of the disease, occur when sickled red blood cells block blood vessels and cause severe pain. Vertex said the mean age of death for patients with sickle cell disease in Europe is about 40 years.
Transfusion-dependent beta thalassemia is a life-threatening genetic blood disorder that requires frequent blood transfusions and iron chelation therapy. Patients may experience anemia-related fatigue, shortness of breath and other complications, including enlarged organs, bone changes and delayed puberty. Vertex said the mean age of death for patients with the disease in Europe is 50 to 55 years.
CASGEVY is approved for eligible sickle cell disease and transfusion-dependent beta thalassemia patients ages 12 and older by multiple regulatory bodies. In the European Union, it is approved for patients ages 12 and older with either severe sickle cell disease with recurrent vaso-occlusive crises or transfusion-dependent beta thalassemia, when hematopoietic stem cell transplantation is appropriate and a matched related donor is not available.
