A fortune is paid for a syringe containing this active ingredient.
On Friday, the European Medicines Agency (EMA) approved conditional approval of the world’s most expensive drug to date.
This product is a drug from the American manufacturer CSL Behring and is intended to help adults against severe and moderate hemophilia B (a bleeding disorder).
A single dose costs lakhs
Dubbed “HemGenix”, the treatment only requires one dose – but that alone costs US$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, there is an inherited defect in blood clotting. The disease is extremely rare, affecting one in 20,000 to 50,000 newborn boys, according to the EMA.
First gene therapy drug for hemophilia
Existing drugs are aimed at improving blood clotting as a preventive measure or in severe cases. But they must be administered repeatedly. “Hemgenics”, on the other hand, causes the liver to start producing the missing substances for blood clotting.
According to the EMA, “HemGenix” is the first gene therapy against the disease. The EU Commission must now decide on an EU-wide conditional market authorization.
Conditional approval may be granted based on less data than usual when there is a specific need for a drug. The manufacturer must then submit additional data.
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