The results of clinical trials conducted in the United States to treat patients with sickle cell disease with a treatment based on Crispr-Cas9 genome modification technology give rise to hope. This hereditary blood disease, particularly debilitating and hitherto incurable, affects several million people worldwide. But not everyone will be able to benefit from this therapeutic innovation, as its cost is high.
The first patient to have successfully benefited from this new gene therapy, Victoria Gray, 37, mother of four children, is the symbol of this medical breakthrough. Fruit of the collaboration started in 2015 between the American Vertex, renowned for having developed Kaftrio, a drug against cystic fibrosis, and the American-Swiss biotech Crispr Therapeutics, this promising therapy is administered in a single injection. On condition that the patient previously undergoes heavy chemotherapy and that a compatible donor is selected.
The two laboratories are now waiting for the precious sesame of the American health authority, the Food and Drug Administration, to be able to market their novelty. If approved, it would become the first gene therapy based on Crispr technology to be licensed in the world. And also one of the most expensive treatments on the market.
If the two laboratories have not yet revealed the selling price of their therapy, experts in the sector estimate that it should exceed one million euros. This is of concern to States, which are increasingly concerned about the ability of their health systems to financially support the arrival of such treatments. Because the drug developed by Vertex and Crispr Therapeutics will not be the first to cross the symbolic threshold of one million euros.
In 2019, the marketing of Zolgensma, an injection worth more than two million dollars (1.8 million euros) intended to cure spinal muscular atrophy, a rare, often fatal neuromuscular disease, had already caused a global shock wave. . Western health systems had jumped in the face of the bill, but still felt they could take the hit (and the cost) due to the very small number of patients concerned. The recent arrival of expensive gene therapies for pathologies involving much larger populations of patients upsets this equation. Like Hemgenix, a treatment against hemophilia labeled in the United States at 3.5 million dollars…
You have 58.91% of this article left to read. The following is for subscribers only.