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

Washington – U.S. health officials on 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 approved Hemgenix, an IV treatment for adults with hemophilia B, the less common form of the genetic disorder that primarily affects men.

Currently, patients receive frequent, expensive IVs of a protein that helps clot and prevent bleeding.

Inside CSL Behring's Privigen Immunoglobulin Production Facility
Signage for CSL Behring, a unit of CSL Ltd., is displayed at one of the company’s factories in Melbourne, Australia, in this July 2, 2014, file photo.

Carla Gottgens/Bloomberg via Getty

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 fewer bleeding events and need fewer clotting treatments.

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

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Like most drugs in the United States, the majority of the cost of the new treatment will be paid by insurance companies, not patients, including private plans and public programs.

After decades of research, gene therapies have begun to reshape the treatment of cancers and rare inherited diseases with drugs that can modify 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 therapy for the more common form of the disorder, hemophilia A.

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

The board has not indicated how long the treatment works. But CSL Behring said patients should benefit from 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 once or more a week to prevent severe bleeding. Untreated, the condition can cause bleeding that seeps into joints and internal organs, including the brain.

Hemgenix delivers a functioning 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 those with the disease, according to the FDA.

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

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

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

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