The FDA (the US government agency that deals with the regulation of food and pharmaceutical products), has approved a $3.5 million drug per dose, now the most expensive in the world. It is the first gene therapy for hemophilia, a one-time treatment for the blood clotting disorder.
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Currently patients receive frequent and expensive drips of a protein that helps clotting and prevents bleeding. Hemgenix, licensed by the Food and Drug Administration, is a treatment for adults with hemophilia B, the least common form of the genetic disorder that primarily affects men. Pharmaceutical maker CSL Behring announced the $3.5 million price tag shortly after the FDA greenlighted it, saying its drug will ultimately reduce healthcare costs because patients will have fewer bleeding episodes and need less treatment.
Like most drugs in the United States, much of the cost of the new treatment will be paid by insurers, not patients, according to media reports. “Today’s approval offers a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies,” said Peter Marks of the FDA. According to the government agency, haemophilia B affects about one in 40,000 people and accounts for about 15% of those affected by the disease.
Last updated: Thursday 24 November 2022, 19:23
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