The European Commission has conditionally authorized the marketing of Casgevy, a gene therapy utilizing CRISPR/Cas9 technology. Developed by Vertex Pharmaceuticals Inc., Casgevy is intended for patients aged 12 and older suffering from aggressive sickle cell anemia. This symptomology may include frequent vaso-occlusive episodes or beta thalassemia that requires regular transfusions, but for which suitable hematopoietic stem cell donors aren't available.
The manufacturer projects that over 8,000 patients aged 12 and above, with severe sickle cell disease or beta thalassemia dependent on transfusion, could become candidates for this treatment. Vertex has already arranged preliminary access for suitable beta thalassemia patients in France, even before the national reimbursement scheme takes effect. The company is also working with hospitals skilled in stem cell transplantation to build a network of independent authorized treatment centers (ATC) for administering Casgevy. Currently, there are three nationally active ATCs, with plans to establish around 25 centers throughout Europe.
In December 2023, Vertex received a positive response from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) regarding the conditional approval of Casgevy. This gene-edited therapy is seen as a groundbreaking advancement in the treatment of severe sickle cell disease and beta thalassemia that is transfusion-dependent. Visit rttnews.com for more health-related news.
