US Approves World’s Most Expensive Drug Worth $3.5 Million a Dose for Hemophilia; All You Need to Know

The US regulators have approved a $3.5 million medicine for haemophilia, making it the most expensive medicine in the world.

The CSL Behring’s haemophilia B gene therapy can free patient from regular treatments but a single dose would cost at $3.5 million.

The CSL Behring’s Hemgenix, can reduce the bleeding events over the course of year by 54 percent if administered once, according to a study.

The medicine will substitute 94 percent of the patients from time-consuming and costly infusions of Factor IX, which is currently used to control the potentially deadly condition, a report in Bloomberg said.

“While the price is a little higher than expected, I do think it has a chance of being successful because 1) existing drugs are also very expensive and 2) hemophilia patients constantly live in fear of bleeds,” said Brad Loncar, a biotechnology investor and chief executive officer of Loncar Investments.

“A gene therapy product will be appealing to some,” Loncar added.

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

The therapy can dramatically improve a range of devastating conditions by fixing their underlying causes. The agency did not specify how long the treatment works. But CSL Behring said patients should benefit— in terms of reduced bleeding and increased clotting — for years.

Earlier in 2019, Novartis AG’s Zolgensma for babies was approved with spinal muscular atrophy was priced at $2.1 million. Bluebird Bio Inc’s Zynteglo for the blood disorder beta thalassemia came in at $2.8 million this year.

Hemophilia almost always strikes males and is caused by mutations in the gene for a protein needed for blood clotting. Small cuts or bruises can be life-threatening, and many people need treatments once or more a week to prevent serious bleeding. Left untreated, the condition can cause bleeding that seeps into joints and internal organs, including the brain.

Hemgenix delivers a working gene for the clotting protein to the liver, where it is made.

Hemophilia B affects about 1 in 40,000 people and accounts for roughly 15% of those with the disease, according to the FDA.

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