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 intravenous treatment for adults with hemophilia B, the rarer form of the genetic disease that mostly affects men. Currently, patients receive frequent, expensive intravenous injections of a protein that helps blood clot and prevents bleeding.
Drugmaker CSL Behring announced the $3.5 million price tag shortly after FDA approval, saying its drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments. The cost appears to exceed that of several other gene therapies costing more than $2 million.
Like most drugs in the US, 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 revolutionize the treatment of cancer and rare inherited diseases with drugs that can modify or correct mutations built into 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 disease, hemophilia 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 does not specify how long the treatment works. 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 a gene for a protein needed 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. If left untreated, the condition can cause bleeding that infiltrates the 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 approximately 15 percent 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 a clotting protein, reduced need for standard treatment and a 54 percent 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 review by the FDA.
___
The Associated Press Health and Science Division is supported by the Howard Hughes Medical Institute Division of Science Education. AP is solely responsible for all content.
Our new weekly Impact Report will explore how ESG news and trends are shaping the roles and responsibilities of today’s executives – and how they can best meet these challenges. Subscribe here.