FDA approves $3.5 million per dose gene therapy for hemophilia B, most expensive drug ever


Washington – US health regulators on Tuesday approved the first gene therapy for hemophilia. The U.S. Food and Drug Administration has approved Hemgenix, his IV treatment for adults with hemophilia B.

Currently, patients frequently receive expensive IVs for proteins that help blood clot and prevent bleeding.

Pennsylvania-based drug company CSL Behring unveiled a $3.5 million price tag shortly after FDA approval that could ultimately reduce health care costs because patients bleed less and need less coagulation therapy. said.

according to Studies cited by the National Library of Medicineits price makes Hemgenix the world’s most expensive drug, easily topped Novartis Zolgensma Gene therapy for spinal muscular atrophy (SMA) costs about $2 million per dose and is also a single-dose drug.

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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.

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 treatment for hemophilia, and several other pharmaceutical companies are working on gene therapy for the more common form of 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.

Hemophilia occurs most often in men and is caused by mutations in genes for proteins necessary for blood clotting. Small cuts and bruises can be life-threatening, and many people need treatment more than once a week to prevent severe bleeding. If left untreated, bleeding can penetrate the joints and internal organs, including the brain.

Hemgenix delivers functional genes for clotting proteins to the liver, where they are made.

According to the FDA, hemophilia B affects approximately 1 in 40,000 people and accounts for approximately 15% of those affected.

The FDA said it granted approval based on two small studies. The study showed that people taking the drug had increased levels of clotting proteins, reduced need for standard care, and bleeding problems he reduced by 54%.

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.


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Written by Natalia Chi

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