The European Medicines Agency (EMA) recommended this Friday granting a conditional European license for Casgevy, an advanced therapy drug for sickle cell anemia and beta thalassemia, which would be the first available treatment based on gene editing technology.
EMA experts have issued a positive opinion on the benefits of the drug intended to counteract the effects of these two genetic disorders and, if the European Commission gives its approval, Casgevy would be the first available treatment that uses CRISPR/Cas9, a type of novel genome editing technology.
The agency, based in Amsterdam, explained that this treatment is indicated for patients with transfusion-dependent beta thalassemia and for severe sickle cell anemia in patients over 12 years of age, for whom “hematopoietic stem cell transplantation is appropriate and there is no suitable donor available.
“This new therapy can free patients from the burden of frequent transfusions and painful vaso-occlusive crises that occur when sickled red blood cells block small blood vessels, and has the potential to significantly improve their quality of life,” explains the EMA. .
Both disorders are rare inherited diseases caused by genetic mutations that affect the production or function of hemoglobin, the protein found in red blood cells that carries oxygen throughout the body. Both diseases are debilitating and life-threatening.
Casgevy is a unique and personalized treatment that involves mobilizing bone marrow cells from the patient’s blood. CRISPR technology is used in this drug to edit the patient’s own blood stem cells, specifically finding a specific DNA sequence within a cell.
The EMA based its positive recommendation on two ongoing single-arm trials. In the first, with 42 patients aged 12 to 35 years and with beta thalassemia who received a single dose, 39 were transfusion-free for at least one year. The second trial included a group of 29 patients who had sickle cell anemia, and 28 of them had no episodes of vaso-occlusive crises for at least 12 months in a row.
The most common side effects were low white blood cell counts, including febrile neutropenia, low platelet levels, liver disease, nausea, vomiting, headache, and mouth sores. These events are due to the drugs needed for the modified blood cells to engraft and replace the unmodified stem cells.
