2023-12-15 16:26:41
The European Medicines Agency (EMA) has recommended the approval of the first medicine that uses gene editing to treat two rare diseases: transfusion-dependent beta thalassemia and severe sickle cell anemia in patients aged 12 and older, which previously could only be cured with a risky stem cell transplant.
In this way, Europe follows in the footsteps of the United States, where this medication was approved on December 8, Casgevywhich uses CRISPR/Cas9, a tool that allows editing the human genome, removing and adding genes at will.
This new therapy, in the words of the EMA, “can free patients from the burden of frequent transfusions and the painful crises that occur when sickled red blood cells block small blood vessels, and has the potential to significantly improve their quality of life.” life”.
Both thalassemia and sickle cell anemia are two rare diseases hereditary diseases caused by genetic mutations that affect the production or function of the hemoglobin, the protein in red blood cells that transports oxygen throughout the body. Both diseases are debilitating, lifelong, and life-threatening. They can cause stroke, organ damage and pain crises.
«Molecular scissors»
CRISPR gene editing makes it possible to find a specific DNA sequence within a cell. Using “molecular scissors” to make precise cuts, it allows genetic material to be added, removed or altered in that specific place in the cells’ genome.
The EMA based its recommendation on two ongoing single-arm trials in patients from 12 to 35 years old. In the first, 42 patients, including 13 adolescents, with transfusion-dependent beta thalassemia who received a single dose were included in the primary efficacy set. Of these 42 patients, 39 were transfusion-free for at least one year.
In the second trial, 29 patients, including six adolescents, who had severe sickle cell anemia, were included in the primary efficacy set. Of these 29 patients, 28 were free of vaso-occlusive crisis (VOC) episodes for at least 12 consecutive months. Characterized by severe pain and organ damage, VOCs are the leading cause of emergency department visits and hospitalizations for patients with sickle cell anemia. Casgevy was supported by the EMA’s PRIority MEdicines (PRIME) programme, which provides early and strengthened scientific and regulatory support for medicines with particular potential to meet unmet medical needs of patients.
In its global evaluation of the available data, the Committee for Advanced Therapies (CAT), the EMA’s committee of experts for cell- and gene-based medicines in which AEMPS technicians participate, considered that Casgevy’s benefits outweighed potential risks in patients with beta thalassemia and severe sickle cell anemia. The CHMP agreed with the assessment and positive opinion of the CAT and recommended the approval of this medicine.
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