New FDA-Approved Hemophilia Gene Therapy Is World’s Priciest Medicine
Drugmaker CSL Bering says the $3.5 million price tag for its Hemgenix treatment is justified because it would ultimately reduce long-term health care costs associated with treating patients living with hemophilia B.
AP:
$3.5M Gene Therapy For Hemophilia Gets FDA Approval
The Food and Drug Administration cleared Hemgenix, an IV treatment for adults with hemophilia B, the less common form of the genetic disorder which primarily affects men. Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevent bleeding. Drugmaker CSL Behring announced the $3.5 million price tag shortly after the FDA approval, saying its drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments. The price appeared to exceed that of several other gene therapies priced upwards of $2 million. (Perrone, 11/22)
Stat:
FDA Approves A Gene Therapy For Hemophilia B
Hemgenix uses an engineered virus to deliver a functional copy of the gene that encodes for Factor IX. Once inserted, the replacement gene instructs cells to produce Factor IX on their own, which in turn, helps the body control bleeding. Currently, people living with hemophilia B receive regular intravenous infusions of Factor IX, which costs approximately $550,000 to $750,000 annually. The $3.5 million price tag for Hemgenix is justified, CSL Behring said, by eliminating the chronic need for Factor IX replacement therapy. (Feuerstein, 11/22)
Reuters:
Australia's CSL Prices Hemophilia Gene Therapy At $3.5 Million
"We are confident this price point will generate significant cost savings for the overall healthcare system and significantly lower the economic burden of hemophilia B," the company said. The list price is not necessarily what the patients pay for the drug. (Leo and Roy, 11/22)
Bloomberg:
World’s Most Expensive Drug Approved to Treat Hemophilia at $3.5 Million a Dose
“While the price is a little higher than expected, I do think it has a chance of being successful because 1) existing drugs are also very expensive and 2) hemophilia patients constantly live in fear of bleeds,” said Brad Loncar, a biotechnology investor and chief executive officer of Loncar Investments. “A gene therapy product will be appealing to some.” (Cortez, 11/23)
