It is a genetic, chronic disease that only affects men: in hemophilia B, blood clotting is disturbed. According to the European Medicines Agency EMA, it is rare, between 20,000 and 50,000 male newborns are affected. With the first drug based on gene therapy, there is now a marketable drug that can make life easier for patients.
The European Medicines Agency (EMA) recommends conditional approval of what observers say is the world’s most expensive drug to date. The preparation “Hemgenix” from the US manufacturer CSL Behring is used in adults against severe and moderate hemophilia B, the EMA announced on Friday in Amsterdam.
At a high price in the truest sense of the word: the drug called “Hemgenix” from the American manufacturer CSL Behring is expensive. A dose of this gene therapy for the US is given at 3.5 million dollars. According to the scientific journal Nature and other sources, this makes it the most expensive drug in the world today.
benefits
However, the advantage is great. “Hemigenix” only needs to be injected once. Existing drugs are aimed at improving blood clotting as a preventive measure or in acute cases. However, they must be administered repeatedly, writes the EMA. “Hemgenix”, on the other hand, causes the liver to start producing the substances that are lacking for blood clotting itself.
According to the EMA recommendation, only the EU Commission should decide on the conditional EU-wide market approval. Conditional approval may be granted based on less data than usual when there is a specific need for a drug. Subsequently, the manufacturer must provide more data.