World’s most expensive FDA-approved drug costs millions – National

The US Food and Drug Administration (FDA) approved the first gene therapy for hemophilia B in the country on Tuesday. This is a drug called Hemgenix that boasts his one-time treatment for blood clotting disorders.

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

With a single dose of $3.5 million, Hemgenix is ​​the world’s most expensive drug, but drug company CSL Behring says the breakthrough drug will ultimately be a drug that patients will need less treatment. to reduce medical costs.

“We believe this price point will provide significant cost savings across the healthcare system, significantly reducing the financial burden of hemophilia B,” Reuters said.

Hemophilia reduces a person’s ability to prevent bleeding, requiring patients to receive frequent and expensive infusions of factor IX, a protein that promotes blood clotting. Small cuts and bruises can be life-threatening, and if left untreated, bleeding can penetrate joints and internal organs, including the brain. caused.

According to the FDA, hemophilia B is less common, accounting for about 15% of patients. About 1 in 40,000 people have her.

As with most medicines in the United States, most of the costs of new treatments are paid by insurance companies, not by patients. This includes private plans and government programs.

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The FDA said it approved Hemgenix based on two small studies. One showed that his bleeding problems had decreased by 54% over the course of a year, and that the patient’s Factor IX levels had risen. Bloomberg reported that HemGenix has shown that 94% of patients have been freed from the time-consuming and costly blood transfusions to control the disease.

Hemgenix works by delivering genes for clotting proteins to the liver, where patients can produce them themselves.

After decades of research, gene therapy is beginning to reshape treatments for cancer and rare genetic diseases using drugs that can alter or correct mutations embedded in people’s genetic code. Hemgenix was the first drug to treat hemophilia in the United States, and several other pharmaceutical companies are working on gene therapy for the more common disease, hemophilia A.

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

The agency didn’t specify how long the treatment would be effective. said.

Earlier this year, European regulators approved a similar gene therapy for hemophilia A. The drug, from his BioMarin pharmaceutical company, is still under FDA review.

The FDA has approved two gene therapies in the United States, Zynteglo and Skysona, from pharmaceutical company Bluebird Bio. According to Reuters, the Jintegro is priced at $2.8 million, while the SkySona is priced at $3 million.

Their approval was met with concerns from investors about the high price. The biotech company’s shares are up 8% since his first treatment was approved in August.

— with files from The Associated Press

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