Washington – US regulators on Tuesday approved the first hemophilia gene therapy, a $3.5 million one-time treatment for blood clotting disorders. The Food and Drug Administration has approved Hemgenix, an intravenous treatment for adults with haemophilia B, a less common form of the genetic disease that mainly affects men.
Currently, patients often receive expensive injections of protein that helps blood clot and prevents bleeding.
Drugmaker CSL Behring announced a $3.5 million price tag shortly after FDA approval, saying its drug would ultimately lower healthcare costs because patients would have fewer bleeding events and need less clotting treatment.
According study cited by the National Library of Medicinethe price makes Hemgenix the most expensive drug in the world, easy to sprinkle gene therapy for spinal muscular atrophy (SMA), which costs about $2 million per dose and is also a single-dose treatment.
As with most dr*gs in the US, most of the cost of a new treatment will be covered by insurers, not patients, including private plans and government programs.
After decades of research, gene therapies have begun to transform the treatment of c*ncer and rare hereditary diseases with dr*gs that can modify or correct mutations embedded in the human genetic code. Hemgenix is the first such haemophilia drug, and several other drugmakers are working on gene therapies for the more common form of the disease, haemophilia 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 Dr. Peter Marks of the FDA.
The agency did not specify how long the treatment works. But CSL Behring said patients should benefit from reduced bleeding and increased clotting over the 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 serious bleeding. Left untreated, the disease can cause bleeding that spreads to joints and internal organs, including the brain.
Hemgenix delivers a working coagulation protein gene to the liver where it is made.
According to the FDA, hemophilia B affects about 1 in 40,000 people and accounts for about 15% of people with the condition.
The FDA said it granted approval based on two small studies, including one that showed people taking the drug had elevated clotting protein levels, a decreased 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 drugmaker BioMarin, is still under review by the FDA.
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