The U.S. Food and Drug Administration has approved a gene therapy for the treatment of haemophilia B. Hemgenix is priced at $3.5 million (£2.9 million) is a one-time treatment for the blood clotting disorder which primarily affects men. Currently, patients receive frequent, and expensive IVs to prevent bleeding. Medics are hopeful this treatment could end the weekly infusions patients receive.
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Expensive price tag
Hemgenix, produced by Pennsylvania drugmaker, CSL Behring, revealed the $3.5 million price tag after the FDA approval. According to the US National Library of Medicine, this makes the drug priciest in the world, a position previously held by Novartis’ Zolgensma, a gene therapy for the treatment of spinal muscular atrophy (SMA). The single-dose medicine cost around $2 million per dose.
It is hoped that the approval of Hemgenix would help reduce health care hosts as patients would have fewer bleeding incidents, thus requiring fewer blood clotting treatments. The FDA's Dr. Peter Marks said in a statement announcing the approval:
Today’s approval provides a new treatment option for patients with Haemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of haemophilia.
Haemophilia B
Haemophilia is usually an inherited bleeding disorder in which the blood does not clot properly, the Centres for Disease Control and Prevention said. The disorder can lead to spontaneous bleeding as well as bleeding following injuries or surgery. It affects at about 1 out of 40,000 people, mostly striking men. For people with this condition, most cuts and bruises can be life-threatening. Hemgenix, the new gene therapy is meant to be a one-time IV infusion.
Sources used:
Business Insider: The most expensive drug in the world has a $3.5 million price tag. 2 potential patients share their hopes and fears for the one-time treatment.
CBC News: FDA approves most expensive drug ever, a $3.5 million-per-dose gene therapy for hemophilia B
CNN: FDA approves $3.5 million treatment for hemophilia, now the most expensive drug in the world
