The FDA approves the most expensive drug in the world: $3.5 million per dose | Health

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

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

Pharmaceutical company CSL Behring announced the $3.5 million price tag shortly after FDA approval, claims that his drug will ultimately reduce health care costs because patients will have fewer bleeding events and require fewer clotting treatments. The price appeared to exceed that of other gene therapies priced at more than $2 million.

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

After decades of research, gene therapies have begun to reshape the treatment of cancers and rare inherited diseases with drugs that can change or correct mutations embedded in people’s genetic code. 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 disease, hemophilia A.

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

The agency did not specify the duration of treatment. But CSL Behring said 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. Minor cuts or bruises can be life-threatening, and many people need treatments once or more a week to prevent serious bleeding. If left untreated, the disease can lead to bleeding that leaks into the joints and internal organs, including the brain.

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

Haemophilia B affects 1 in 40,000 people and accounts for around 15% of sufferersaccording to the FDA.

The FDA granted approval based on two small studies, one of which showed that those who took the drug had higher levels of clotting proteins, less need for standard treatment and a 54% decrease in bleeding problems.

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

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