Approvals of Hemlibra
Montreal, September 13, 2021 – In recent weeks, governments in both Quebec and the rest of Canada have expanded access to Hemlibra to patients with hemophilia A without inhibitors. Unlike factor VIII concentrates, however, Hemlibra will not be available to all people with hemophilia A.
Hemlibra was initially approved in summer 2019 for all those with inhibitors to factor VIII.
Who then is eligible in the regions served by Canadian Blood Services (CBS)?
In the provinces and territories served by CBS (all except Quebec), health authorities expanded access, without age restrictions, to include those with severe congenital hemophilia A (intrinsic factor VIII level < 1%) without inhibitors who are candidates for routine prophylaxis to prevent bleeding or reduce the frequency of bleeding episodes. This means that those people with moderate hemophilia, even those with factor VIII levels just above 1%, are not eligible. All the research studies leading to the licensure of Hemlibra were carried out in people with severe hemophilia A. A factor VIII level of 1% or less is the standard definition of severe hemophilia. Research is only now starting into the efficacy of Hemlibra in populations with factor VIII levels above 1%.
It is becoming better recognized that factor level, as measured by a lab test, is not the only element affecting the severity of clinical symptoms. Other issues, some understood and others not, affect how often and how easily people bleed. Some people with levels below 1% rarely bleed with no apparent cause, while others in the moderate range of 1-5% can suffer from frequent non-traumatic bleeds. It can only be hoped that as research continues and the tendency to consider clinical bleeding as opposed to factor assays measured by lab tests gains acceptance, patients with moderate hemophilia who require prophylaxis will become eligible.
CBS has announced that Hemlibra should be available in hemophilia centers by mid-October.