San Francisco Chronicle

FDA approves gene therapy

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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 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.

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