The first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting illness, was approved by U.S. health officials on Tuesday.
As per CTV News, The Food and Drug Administration approved Hemgenix, an intravenous medication for adults with hemophilia B, a less prevalent hereditary condition that mostly affects men. Patients already receive frequent, costly intravenous infusions of a protein that promotes blood clotting and prevents bleeding.
Shortly after FDA clearance, the medicine’s manufacturer, CSL Behring, disclosed the drug’s price tag of $3.5 million, stating that the therapy would ultimately save health care expenditures since patients would experience fewer bleeding events and require fewer clotting therapies. The cost looked to exceed that of a number of other gene therapies that cost over $2 million.
As with the majority of medications in the United States, the majority of the cost of the new treatment will be covered by insurers, both commercial plans and government programs.
After decades of research, gene therapies have begun to reshape the treatment of malignancies and uncommon inherited disorders using drugs that can modify or fix mutations in the genetic code of individuals. Hemgenix is the first treatment of its kind for hemophilia, and other pharmaceutical companies are developing gene treatments for the more prevalent form of the illness, hemophilia A.
“Today’s approval gives patients with hemophilia B a new treatment choice and reflects significant progress in the development of innovative medicines,” said Dr. Peter Marks of the FDA.
The agency did not specify the treatment’s duration of effectiveness. However, according to CSL Behring, patients should see reduced bleeding and increased clotting for years.
Hemophilia is caused by abnormalities in the gene for a protein required for blood clotting and almost usually affects males. Small cuts and bruises can be life-threatening, and many patients require weekly or more frequent treatments to prevent significant bleeding. The illness, if left untreated, can cause bleeding into joints and other organs, including the brain.
Hemgenix transports a functional gene for the clotting protein to the liver, where it is synthesized. According to the FDA, hemophilia B affects around 1 in 40,000 people and accounts for approximately 15% of those with the illness.
The FDA stated that it granted approval based on two small studies, including one that demonstrated increased levels of the clotting protein, decreased need for usual treatment, and a 54% decrease in bleeding difficulties among those taking the medicine.
European regulators approved a similar gene treatment for hemophilia A earlier this year. This medicine, manufactured by BioMarin, is still being evaluated by the FDA.
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