The FDA Has Approved The World’s Most Expensive Drug – And It Costs $3.5 Million

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The US Food and Drug Administration (FDA) recently approved a new drug called Hemgenix, it is a medication for a blood clotting disorder (hemophilia B). Hemgenix is a gene therapy to treat adults with hemophilia B, a genetic bleeding disorder in which people do not produce a protein needed to create blood clots. About 1 in 40,000 people have the disease, most of whom are men.

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

Per dose, it’ll cost US$3.5 million, making it the most expensive drug anywhere in the world. At first glance, the price is shocking, but a recent analysis of the cost-effectiveness of the drug suggests that’s a relatively ‘fair’ price for what the treatment achieves – at least in the US.

Like most medicines in the U.S., most of the cost of the new treatment will be paid by insurers — not patients — including private plans and government programs.

The main medication currently used to treat hemophilia B in the US bestows patients with a much-needed clotting factor, but its lifetime treatment costs are steep. In those with severe symptoms, a routine and expensive treatment regime are required, one that over time can begin to wane in effectiveness.

The FDA said it granted approval based on two small studies, including one that showed those taking the drug had increased levels of clotting protein, reduced need for standard treatment and a 54% drop in bleeding problems. Earlier this year, European regulators approved a similar gene therapy for hemophilia A. That drug, from drugmaker BioMarin, is still under review at the FDA.   

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