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Washington — 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, based in Pennsylvania, 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.
According to a study cited by the National Library of Medicine, the price makes Hemgenix the most expensive medicine in the world, easily topping Novartis' Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs right around $2 million per dose and is also a single-dose medicine.
www.cbsnews.com
This is too insane.........
Currently, patients receive frequent, expensive IVs of a protein that helps blood clot and prevent bleeding.
Drugmaker CSL Behring, based in Pennsylvania, 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.
According to a study cited by the National Library of Medicine, the price makes Hemgenix the most expensive medicine in the world, easily topping Novartis' Zolgensma gene therapy for spinal muscular atrophy (SMA), which costs right around $2 million per dose and is also a single-dose medicine.
FDA approves most expensive drug ever, a $3.5 million-per-dose gene therapy for hemophilia B
The single-dose medicine "represents important progress" as the first-ever gene therapy for the rare disorder, the FDA said.
www.cbsnews.com
This is too insane.........
