Hemgenix The most expensive drug in the world: 3,5 million dollars for a dose

US health regulators this week approved the first gene therapy for hemophilia, a one-time, $3,5 million treatment for the blood-clotting disorder.

blood cells

The Food and Drug Administration (FDA) has approved Hemgenix, an intravenous treatment for adults with hemophilia B, the less common form of a genetic disorder that mostly affects men. Currently, patients receive frequent, expensive intravenous doses of a protein that helps blood clot and prevent bleeding.

Pennsylvania-based pharmaceutical company CSL Behring announced the $3,5 million price tag shortly after the FDA approval, saying its drug will ultimately lower health care costs because patients will have fewer bleeding events and fewer doses will be needed for blood clotting.

According to a study by the National Library of Medicine, the price makes Hemgenix the most expensive drug in the world, easily surpassing Novartis' Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs about $2 million per dose and is also single-dose medicine.

Like most drugs in the US, most of the cost of the new treatment will be paid by private or government insurance companies, not patients.

After decades of research, gene therapies have begun to reshape the treatment of cancers and rare inherited diseases with drugs that can modify or correct mutations built into people's genetic code.

Hemgenix is ​​a first such treatment for hemophilia. Several scientists are working on gene therapies for the most common form of the disorder, hemophilia A.

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Hemgenix, expensive drug

Written by giorgos

George still wonders what he's doing here ...

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