CHMP recommends gene therapy for beta thalassemia and sickle cell disease
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel) to treat transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD).
SCD and beta thalassemia are genetic conditions caused by errors in the gene for haemoglobin, which is used by red blood cells to deliver oxygen around the body.
The cell-based gene therapy is the first medicine to use CRISPR/Cas9 to edit the faulty gene in patients’ bone marrow stem cells to allow the body to produce functioning haemoglobin.
If approved, Casgevy would be the only genetic therapy available to EU patients aged 12 and older with either severe SCD with recurrent vaco-occlusive crises (VOC) or TDT for whom hematopoietic stem cell transplantation is appropriate and a suitable donor is not valid.
The recommendation follows the announcement from EMA’s Committee for Advanced Therapies that the benefits of Casgevy outweighed the possible risks in patients with TDT and SCD.
