New most expensive drug in the world: what is it for and why does Hemgenix cost so much

  • Atahualpa Amerise @atareports
  • BBC News World

scientist in laboratory

Credit, Getty Images

The drug Hemgenix recently received approval from the regulatory agency for food and drugs in the United States (FDA, its acronym in English) to be marketed in the country.

It is a gene therapy to treat haemophilia B, the least common and most dangerous form of the disease that makes it difficult for blood to clot.

CSL Behring, the drug’s maker, has priced a single-dose treatment at $3.5 million in the United States — making it by far the most expensive drug in the world.

what is it for

Approximately one in every 40,000 people has hemophilia B, a disease that affects men more, as many women are asymptomatic.

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