World's most expensive drug approved by FDA, treats Hemophilia

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The US Food and Drug Administration (FDA) has approved a drug called Hemgenix, the first gene therapy for hemophilia B in the country, Global News reported on November 23. This drug is a one-time treatment for the blood clotting disorder.

With this, the most expensive drug has now been put for sale in the market by the Agency.

Hemgenix stands to be the costliest drug in the world, priced at $3.5 million per dose. But, drugmaker CSL Behring states that this groundbreaking medicine will come to reducing healthcare-costs, and patients may 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 is a condition that compromises upon the ability of a person to prevent bleeding and requires them to receive multiple and expensive IV drips of Factor IX. This is a protein that primarily encourages blood clotting. For a person suffering from the same, any small cuts or bruises can be life threatening if left untreated. Hemophilia is caused by gene mutation and generally affects men.

Hemophilia B is the lesser common form of the disorder, and accounts for 15 percent of the ones affected by it, as mentioned by FDA. It affects nearly one in 40,000 people.

Hemgenix functions by delivering a certain gene for the clotting protein to the liver, post which the patient can further produce it on their own.