NEW YORK – The European Commission has granted conditional marketing authorization to Vertex Pharmaceuticals' Casgevy (exagamglogene autotemcel) as a treatment for sickle cell disease and transfusion-dependent beta thalassemia, the biotech firm said Tuesday.
Casgevy, developed by Vertex and CRISPR Therapeutics, is the first CRISPR-based therapy approved in the EU, a milestone the gene-editing therapy also met in the US and UK, where it has conditional marketing authorization.
In the EU, regulators may grant conditional authorization to treatments that fulfill a significant unmet medical need, but for which comprehensive clinical evidence of efficacy and safety is not yet available. Conditional marketing authorizations are valid for one year and can be renewed annually based on an ongoing regulatory review of the company's data.
As such, Casgevy will be available to patients at least 12 years of age who have severe sickle cell disease with recurrent vaso-occlusive crises and to transfusion-dependent beta thalassemia patients who aren't eligible for hematopoietic stem cell transplantation.
Vertex said it is working with health authorities in specific EU countries to secure access for eligible patients. For example, it has established early access for patients in France with transfusion-dependent beta thalassemia ahead of the national reimbursement process.
"With this approval, Casgevy is now approved for sickle cell disease and transfusion-dependent beta thalassemia in multiple geographies making tens of thousands of patients eligible for this potentially transformative therapy," Vertex CEO and President Reshma Kewalramani said in a statement. "Now our goal shifts to translating these approvals into real-world patient benefit and ensuring access and reimbursement across the globe."
Vertex plans to have a network of 25 sites that serve as authorized treatment centers to administer the therapy in Europe. So far, the firm has set up three such centers.
Patients with sickle cell and transfusion-dependent beta thalassemia have deficiencies in the adult version of hemoglobin. Treatment with Casgevy involves editing patients' own CD34-positive hematopoietic stem and progenitor cells ex vivo with CRISPR-Cas9. Specifically, the BCL11A gene is edited to produce fetal hemoglobin, which then facilitates oxygen delivery.