This time, it’s done ! The first treatment designed using the Crispr genome editing system has just been approved by the European Medicines Agency (EMA). On Friday, December 15, the health authority asked the European Commission to officially authorize Casgevy for patients over 12 years of age suffering from severe forms of sickle cell anemia or beta thalassemia, two particularly disabling blood pathologies of genetic origin. .
This step being a simple formality, it will then be up to the Member States to determine the conditions of use of the treatment and its cost. But after the green light granted on November 16 by the British health agency and that delivered on December 8 by the American Food and Drug Administration, the start-ups Vertex Pharmaceuticals and Crispr Therapeutics which designed the product can congratulate themselves on having was a hit.
With 300,000 to 400,000 children born each year with this pathology, sickle cell disease is the most widespread monogenic disease in the world. In the blood of affected people, the red blood cells take on the shape of sickles, hence its other name, sickle cell anemia. Not only do blood cells no longer transport oxygen as well, but they tend to clump together and create clots. In addition to chronic anemia, which requires transfusions, patients then suffer from painful crises which can also lead to the deterioration of various organs, strokes and early death.
Moderate side effects
During the last International Summit on Genome Editing, held in London from March 6 to 8, the American Victoria Gray, 37, shocked the audience by recounting the multiple hospitalizations and the intense suffering that had punctuated his life since childhood. The young mother had especially insisted on the way in which the treatment, of which she had been the first beneficiary, three years previously, had transformed her life: “I am proof that miracles can happen”she declared.
The results of the clinical trial, recalled by the EMA, are, it is true, impressive. Twenty-nine patients between 12 and 35 years old, including 6 adolescents, all suffered from severe forms of sickle cell disease. Among them, 28 no longer endured, for at least twelve months, any of the terrible vaso-inclusive crises from which they suffered and which led them to hospital.
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