FDA clears first gene therapy for hemophilia: CSL Behring’s $3.5 million Hemgenix


US health regulators on Tuesday approved the first gene therapy for hemophilia, a one-time $3.5 million treatment for the blood-clotting disorder.

The Food and Drug Administration has approved Hemgenix, an intravenous treatment for adults with hemophilia B, the less common form of the genetic disorder that primarily affects men. Currently, patients receive frequent and expensive intravenous lines of a protein that helps blood clot and prevents bleeding.

Drugmaker CSL Behring announced the $3.5 million price tag shortly after FDA approval, saying its drug would ultimately lower healthcare costs because patients would have fewer bleeding incidents and need fewer treatments. of coagulation. The price appeared to exceed that of several other gene therapies with prices higher than $2 million

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

After decades of research, gene therapies have begun to reshape cancer treatment and rare hereditary diseases with drugs that can modify or correct mutations embedded in the genetic code of people. Hemgenix is ​​the first treatment of its kind for hemophilia, and several other drugmakers are working on gene therapies for the most 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 the FDA’s Dr. Peter Marks.

The agency did not specify how long the treatment works. But CSL Behring said that patients should benefit, in terms of reduced bleeding and increased clotting, for years.

Hemophilia almost always affects men and is caused by mutations in the gene for a protein needed for blood clotting. Small cuts or bruises can be life-threatening, and many people need treatments one or more times a week to prevent serious bleeding. If left untreated, the condition can cause bleeding to seep into the joints and internal organs, including the brain.

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

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

The FDA said it granted the approval based on two small studies, including one that showed those taking the drug had elevated levels of clotting protein, a reduced need for standard treatment, and a 54% reduction in bleeding problems.

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


The Associated Press Department of Health and Science receives support from the Howard Hughes Medical Institute Department of Science Education. The AP is solely responsible for all content.

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