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

Washington— U.S. 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 IV treatment for adults with hemophilia B, the least common form of the genetic condition that primarily affects men.

Currently, patients receive frequent and expensive intravenous injections of a protein that helps blood clot and prevents bleeding.

Pennsylvania-based drugmaker CSL Behring announced the $3.5 million price tag shortly after FDA approval, saying its drug would ultimately reduce healthcare costs because patients would have less bleeding events and would need fewer coagulation treatments.

According to a study cited by the National Library of Medicine, the price makes Hemgenix the most expensive drug in the world, easily exceeding Zolgensma by Novartis gene therapy for spinal muscular atrophy (SMA), which costs about $2 million per dose and is also a single-dose drug.


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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 capable of altering or correcting mutations embedded in people’s genetic code. Hemgenix is ​​the first of its kind treatment 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 significant progress in the development of innovative therapies,” said FDA’s Dr. Peter Marks.

The agency did not say how long the treatment works. But CSL Behring said patients should benefit from reduced bleeding and increased clotting for years.

Hemophilia almost always strikes men and is caused by mutations in the gene for a protein necessary for blood clotting. Small cuts or bruises can be life-threatening, and many people need treatment once or more a week to prevent serious bleeding. Untreated, the disease can cause bleeding that seeps into joints and internal organs, including the brain.

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

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 its approval based on two small studies, including one that showed those taking the drug had increased levels of clotting protein, a reduced need for standard treatment and a 54% drop in problems. of bleeding.

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

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