This time it's done! The first treatment developed using the Crispr genome editing system has just been approved by the European Medicines Agency (EMA). On Friday, December 15, the health agency called on the European Commission to formally approve Casgevy for patients over 12 years old who suffer from severe forms of sickle cell anemia or beta-thalassemia, two particularly disabling blood diseases of genetic origin.
Since this step is a simple formality, it is then up to the Member States to determine the conditions for receiving the treatment and its costs. But after the British health authority gave the green light on November 16th and the US Food and Drug Administration (FDA) gave the green light on December 8th, the start-ups Vertex Pharmaceuticals and Crispr Therapeutics, which developed the product, can congratulate themselves on this success.
Also read: A first Crispr drug approved in the USA
With 300,000 to 400,000 children born with this pathology each year, sickle cell anemia is the most widespread monogenic disease worldwide. In the blood of those affected, the red blood cells take the shape of sickles, hence the other name, sickle cell anemia. Not only do blood cells no longer transport oxygen as well, they also tend to clump together and form blood clots. In addition to chronic anemia, which requires transfusions, painful crises occur, which can also lead to the deterioration of various organs, strokes and early death.
Moderate side effects
During the recent International Summit on Genome Editing, held in London from March 6 to 8, 37-year-old American Victoria Gray shocked the audience by recounting the numerous hospitalizations and intense suffering that have characterized his life since childhood had shaped. The young mother had particularly emphasized how the treatment from which she had first benefited three years earlier had changed her life: “I am proof that miracles can happen,” she explained.
The results of the clinical study recalled by the EMA are impressive. 29 patients between the ages of 12 and 35, including six teenagers, all suffered from severe forms of sickle cell anemia. Of them, 28 did not endure the terrible vascular crises that they suffered and that landed them in the hospital for at least 12 months.
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