MOSCOW, Nov 16 The Medicines and Healthcare Products Administration in the UK has approved the use of the world's first gene therapy for the treatment of sickle cell anemia and beta thalassemia, the government website reports.
“The Drug and Healthcare Products Administration, after a thorough evaluation of the safety, quality and effectiveness of the new drug, has approved its use for the treatment of sickle cell disease and transfusion-dependent beta thalassemia for patients aged 12 years and older,” the statement said.
According to the regulator, Casgevy is the first licensed drug that uses the innovative gene editing tool CRISPR, for the creation of which its inventors were awarded the Nobel Prize in 2020. Sickle cell anemia and beta thalassemia are genetic diseases caused by errors in the hemoglobin genes.
Around 15,000 people in the UK have sickle cell disease. Patients with beta thalassemia require blood transfusions every 3-5 weeks. The use of therapy can save the patient from the need for ongoing treatment for many years, potentially for life. As a result of testing the drug, 97% of patients (28 people) got rid of attacks of pain that accompany the disease for a year, the material says.
Tests on the use of the drug are ongoing.
Casvegy is used by taking stem cells from the bone marrow and editing the gene in those cells in laboratories. Patients must then undergo a course of symptomatic treatment, after which the modified cells are reintroduced into the body. After this, the patient may have to spend at least a month in the hospital until the changed cells take root in the bone marrow and begin to produce red blood cells with a stable form of hemoglobin.