Roche’s Hemlibra will get a speedy review in the US for adults and children with haemophilia A without factor VIII inhibitors.
The submission is based on data from 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.
“People with haemophilia A can face significant challenges in managing their condition and may need to adapt their daily lives to avoid bleeds and accommodate treatment,” said Sandra Horning, Roche’s chief medical officer and head of Global Product Development.
“We believe the FDA’s decision to grant Priority Review to Hemlibra underscores its potential to improve the standard of care for people without factor VIII inhibitors and to help reduce treatment burden by offering more flexible subcutaneous dosing options.”
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.