US health regulators on Tuesday approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder. The FDA cleared Hemgenix: It's an IV treatment for adults with hemophilia B, the less common form of the genetic disorder, reports the AP. Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevents 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 upward of $2 million. Indeed, CBS News reports a December 2021 study cited by the National Library of Medicine noted that Novartis' Zolgensma gene therapy for spinal muscular atrophy was the most expensive medicine in the world at $2 million a dose, suggesting Hemgenix is now the most expensive FDA-approved drug. Bloomberg agrees, calling it "the most expensive medicine in the world." Like most medicines in the US, most of the cost of the new treatment will be paid by insurers, not patients. The agency didn't specify how long the treatment works. But CSL Behring said patients should benefit—in terms of reduced bleeding and increased clotting—for years.
After decades of research, gene therapies have begun reshaping the treatment of cancers and rare inheritable diseases with medicines that can modify or correct mutations embedded in people's genetic code. Hemgenix is the first such treatment for hemophilia, which almost always strikes males 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. Hemgenix delivers a working gene for the clotting protein to the liver, where it's made. Several other drugmakers are working on gene therapies for the more common form of the disorder, hemophilia A.
(More
hemophilia stories.)
,
uBlock 
,
and
Ghostery 
)
