World’s most expensive drug approved by FDA has multimillion-dollar price tag

Hemgenix, by drugmaker CSL Behring, boasts a one-time treatment for hemophilia B, a less common form of the blood-clotting disorder.

The U.S. Food and Drug Administration (FDA) approved the first gene therapy for hemophilia B in the country on Tuesday — a drug called Hemgenix that boasts a one-time treatment for the blood-clotting disorder.

In doing so, the agency has put the most expensive medicine ever on the market.

At US$3.5 million per dose, Hemgenix stands as the priciest drug in the world, but drugmaker CSL Behring says the groundbreaking medicine will ultimately reduce health-care costs because patients would require fewer treatments.

“We are confident this price point will generate significant cost savings for the overall health-care system and significantly lower the economic burden of hemophilia B,” the company said, according to Reuters.

Hemophilia compromises a person’s ability to prevent bleeding and requires patients to receive frequent and expensive IV drips of Factor IX, a protein that encourages blood clotting. Small cuts or bruises can be life-threatening, and if left untreated, the condition can cause bleeding that seeps into joints and internal organs, including the brain. Hemophilia primarily affects men and is caused by a gene mutation.

Hemophilia B is the less common form of the disorder, accounting for roughly 15 per cent of those with the disease, according to the FDA. About one in 40,000 people is affected by it.

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 FDA said that it approved Hemgenix based on two small studies. One showed a 54 per cent drop in bleeding problems over the course of a year and increased patients’ levels of Factor IX. Bloomberg reported that Hemgenix was shown to free 94 per cent of patients from time-consuming and costly transfusions to control the disease.