The price for a dose of the gene therapy “Hemgenix” from the American manufacturer CSL Behring – the drug only needs to be injected once – is given for the US at $ 3.5 million. According to the scientific journal “Nature” and other sources, it is currently the most expensive drug in the world.
In hemophilia B, blood clotting is hereditary. The disease is very rare, affecting about one in 20,000 to 50,000 male newborns, according to the EMA.
Existing drugs are aimed at improving blood clotting, either preventively or in acute cases. However, they must be administered again and again, the EMA writes. “Hemgenix”, on the other hand, causes the liver to start producing the missing substances for blood clotting on its own.
“Hemgenix” is the first gene therapy against the disease, according to the EMA. The EU Commission must now decide on the EU-wide conditional market approval.
Conditional approval can be granted on the basis of less data than usual when there is a specific need for a medicine. The manufacturer should provide more details later.
(SDA)