For the first time, the European Medicines Agency Ema recommends the conditional approval of a therapy based on the Crispr genetic scissors technology. “Casgevy” is suitable for the treatment of sickle cell disease and beta thalassemia in patients aged twelve and over, said Ema in Amsterdam.
Hereditary blood diseases are caused by genetic errors that affect the formation or functioning of hemoglobin. Hemoglobin is an iron-containing protein complex found in red blood cells and used to transport oxygen.
The conditional approval is intended to make the drug quickly available to patients because of its high benefit. Ema bases the recommendation of “Casgevy” on two studies and a long-term follow-up study. The manufacturer must provide results of further studies by 2026 to prove the effectiveness and safety of the drug. The EU Commission now has to agree to EU-wide market approval. Great Britain had already approved “Casgevy” in mid-November this year.
The so-called Crispr/Cas gene scissors can be specifically targeted at individual genes. The developers of the method, Emmanuelle Charpentier and Jennifer A. Doudna, received the Nobel Prize for this in 2020. “Casgevy” is used to change genes in patients’ bone marrow stem cells so that they produce functioning hemoglobin again. To do this, stem cells are taken from the bone marrow, processed in the laboratory and then reinserted into the patient.
Sickle cell disease can cause severe attacks of pain, serious and life-threatening infections, and anemia, a lack of oxygen in the blood also known as anemia. Patients with beta thalassemia also suffer from anemia and often require blood transfusions at intervals of three to five weeks. Until now, a bone marrow transplant was considered the only permanent treatment option.
