The committee that evaluates medicinal products for human use (CHMP) at the European Medicines Agency (EMA) has recommended the authorization of Casgevy, the first drug based on CRISPR technologythe genetic editing tool that allows you to ‘cut and paste’ DNA.
This is not a definitive authorization. In order for the product to be marketed in the European Union, it must first receive authorization from the European Commission, which, however, usually follow the committee’s recommendations.
Advanced therapy would be indicated for the treatment of sickle cell disease (also called sickle cell disease) and transfusion-dependent beta-thalassemiatwo rare diseases caused by genetic mutations that affect the production and functioning of hemoglobin, the protein found in red blood cells that is responsible for transporting oxygen throughout the body.
Specifically, the committee indicates that the therapy could be applied for the treatment of transfusion-dependent beta-thalassemia in patients 12 years or older for whom hematopoietic stem cell transplantation (HSCT) is appropriate and a suitable donor is not available.
In the case of sickle cell disease, the indication would be for those over 12 years of age who present recurrent vaso-occlusive crises. One of the main characteristics of the disease is that, due to the characteristics of their red blood cells, those affected frequently suffer these acute pain crises.