$3.5m Gene Therapy for Hemophilia Gets FDA Approval

U.S. health regulators approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder.

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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.

Like most medicines in the U.S., most of the cost of the new treatment will be paid by insurers not patients including private plans and government programs.