Washington — U.S. health regulators 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 has 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 blood to clot and prevent bleeding.
Pennsylvania-based drugmaker CSL Behring announced the $3.5 million price tag shortly after FDA approval, saying the drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments. to have.
According to an study cited by the National Library of Medicinethe price makes Hemgenix the most expensive drug in the world, easily surpassed gene therapy for spinal muscular atrophy (SMA), which costs about $2 million per dose and is also a single-dose drug.
Like most drugs in the US, most of the cost of the new treatment will be paid for by insurers, not patients, including private plans and government programs.
After decades of research, gene therapies have begun to revolutionize the treatment of cancer and rare hereditary diseases with drugs that can modify or correct mutations embedded in people’s genetic code. Hemgenix is the first such treatment for hemophilia, and several other drugmakers are working on gene therapies for the more common form of the condition, hemophilia A.
“Today’s approval provides a new treatment option for patients with hemophilia B and represents an important advancement in the development of innovative therapies,” said Dr. Peter Marks of the FDA.
The agency has not specified how long the treatment works. But CSL Behring said patients should benefit for years in terms of reduced bleeding and increased clotting.
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 require one or more treatments per week to prevent serious bleeding. Left untreated, the condition can cause bleeding that seeps into joints and internal organs, including the brain.
Hemgenix delivers a working 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, less need for standard treatment and a 54% drop in bleeding problems.
Earlier this year, European regulators approved a similar gene therapy for hemophilia A. That drug, from drugmaker BioMarin, is still under FDA review.