Wed. Nov 23rd, 2022

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

With this, the agency put the most expensive drug ever on the market.

At US$3.5 million per dose, Hemgenix is ​​the most expensive drug in the world, but drugmaker CSL Behring says the breakthrough drug will ultimately reduce healthcare costs because patients will need fewer treatments.

“We are confident that this price will generate significant savings for the entire healthcare system and significantly reduce the economic burden of hemophilia B,” the company said, according to Reuters.

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

Hemophilia B is a rarer form of the disorder and accounts for approximately 15 percent of those affected, according to the FDA. About one in 40,000 people are affected by it.

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Like most drugs 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 it approved Hemgenix based on two small studies. One showed a 54 percent reduction in bleeding problems over a year and an increase in patients’ factor IX levels. Bloomberg reported that Hemgenix has been shown to free 94 percent of patients from long-term and expensive transfusions to control the disease.

Hemgenix works by delivering the gene for the clotting protein to the liver, where the patient can produce it himself.

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After decades of research, gene therapies have begun to reshape the treatment of cancer and rare inherited diseases with drugs that can modify or correct mutations built into people’s genetic code. Hemgenix is ​​the first such hemophilia drug in the U.S., and several other drugmakers are working on gene therapies for the common form of the disorder, hemophilia A.

“Today’s approval provides a new treatment option for patients with hemophilia B and represents an important advance in the development of innovative therapies,” said Dr. Peter Marks of the FDA.

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.

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Earlier this year, European regulators approved a similar gene therapy for hemophilia A. That drug, from drugmaker BioMarin, is still under FDA review.

The FDA already has two gene therapies approved in the US, Zynteglo and Skyson by drugmaker Bluebird Bio. Reuters reports that Zyntegl is priced at $2.8 million and Skyson at $3 million.

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Their approvals have been met with investor concerns over high prices. Shares in the biotech company have risen eight percent since the approval of the first treatment in August.

— With files from the Associated Press

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