Roche has picked up US approval for Hemlibra to prevent or reduce bleeding episodes in patients with haemophilia A without factor VIII inhibitors.
Hemlibra is now the only prophylactic treatment for people with haemophilia A with and without factor VIII inhibitors that can be administered subcutaneously and at multiple dosing options.
The approval is based on positive results from the phase III HAVEN 3 and HAVEN 4 studies.
Hemlibra prophylaxis led to statistically significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis (primary endpoint) and across all other bleed-related endpoints in the HAVEN 3 study and showed a clinically meaningful control of bleeding in the HAVEN 4 study.
“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, haematologist at the 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.
“Hemlibra can reduce bleeds, and it offers a new subcutaneous administration once weekly, every two weeks or every four weeks.”
Hemlibra was granted Breakthrough Therapy Designation by the FDA for haemophilia A without factor VIII inhibitors. It was also granted Priority Review.