Roche’s Hemlibra has been approved in the US for routine prophylaxis of bleeding episodes patients with haemophilia A without factor VIII inhibitors.
According to Roche, Hemlibra is the only medicine that can be self-administered subcutaneously once weekly, every two weeks or every four weeks for haemophilia A with and without factor VIII inhibitors.
“Many preventative treatment options for people with haemophilia A without factor VIII inhibitors require intravenous infusions several times a week. Even then, people can still experience bleeds, and there has been a need for more treatment options,” said Michael Callaghan, MD, haematologist, Children’s Hospital of Michigan, Detroit.
“The approval of Hemlibra is an important advancement for the entire haemophilia A community, as we now have a new class of medicine for the first time in nearly 20 years.”
The decision follows results of the Phase III HAVEN 3 study, in which adults and adolescents aged 12 years or older without factor VIII inhibitors who received Hemlibra prophylaxis every week or every two weeks showed a 96 percent and 97 percent reduction in treated bleeds, respectively, compared to those who received no prophylaxis.
In addition, 55.6 percent of people treated with Hemlibra every week and 60 percent of those given the drug every two weeks experienced zero treated bleeds, compared to 0 percent of people treated with no prophylaxis.
On the safety side, there were no unexpected or serious adverse events (AEs) related to the therapy observed in the study, the most common being injection site reactions, joint pain, common cold symptoms, headache, upper respiratory tract infection and influenza.
Hemlibra was first cleared by the FDA in November 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with haemophilia A with factor VIII inhibitors based on results from the HAVEN 1 and HAVEN 2 studies.
The drug is also approved in the EU for the same indication, and is currently being reviewed for patients without factor VIII inhibitors.
