Hemgenix is the first and only onetime gene therapy treatment for adults with hemophilia B who “currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes,” according to a Nov. 22 CSL release.
The drug reduces the rate of annual bleeds, reduces or eliminates the need for prophylactic therapy and generates elevated and sustained factor IX levels for adults with hemophilia B, according to the release. Ninety-four percent of patients discontinue factor IX prophylaxis and remained prophylaxis-free after treatment, the release stated.
The drug will be commercialized immediately at the price point of $3.5 million. According to a CSL statement, this will “generate significant cost savings for the overall healthcare system and significantly lower the economic burden of hemophilia B.”
The company estimates that people with moderate-to-severe hemophilia B can accumulate $20 million in treatment over a lifetime.
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