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

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US regulators have approved a $3.5 million drug for hemophilia, making it the most expensive drug in the world.

CSL Behring’s hemophilia B gene therapy may free the patient from regular treatments, but a single dose would cost $3.5 million.

According to one study, CSL Behring’s Hemgenix can reduce bleeding events by 54% over the year when given just once.

The drug will replace 94% of patients with long and expensive infusions of factor IX, which is currently used to control the life-threatening disease, according to a Bloomberg report.

“Although the price is a bit higher than expected, I think it has a chance of succeeding because 1) existing drugs are also very expensive and 2) hemophilia patients live in constant fear of bleeding,” said Brad Loncar, a biotechnology investor. and Managing Director of Loncar Investments.

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

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

Therapy can dramatically improve a range of devastating conditions by correcting their underlying causes. The agency did not say how long the treatment works. But CSL Behring said patients stand to benefit – in terms of reduced bleeding and increased clotting – for years.

Earlier in 2019, Novartis AG’s baby Zolgensma was approved with spinal muscular atrophy at a price of $2.1 million. Bluebird Bio Inc’s Zynteglo for the blood disorder beta-thalassemia cost $2.8 million this year.

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

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

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

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