U.S. health regulators have approved the first gene therapy for hemophilia, a blood-clotting disorder with few treatment options.
FILE - The U.S. Food and Drug Administration building stands behind an FDA logo at a bus stop on the agency's campus on Aug. 2, 2018, in Silver Spring, Md. The U.S. health regulators on Tuesday, Nov. 22, 2022, have approved the first gene therapy for hemophilia, a $3.5 million treatment that paves the way for new options in treating the blood-clotting disorder. – U.S. health regulators on Tuesday approved the first gene therapy for hemophilia, a $3.
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.rare inheritable diseases The agency did not specify how long the treatment works. But CSL Behring said patients should benefit— in terms of reduced bleeding and increased clotting — for years.
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$3.5M gene therapy for hemophilia gets FDA approvalU.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. 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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