FDA approves Roche’s Hemlibra for hemophilia A without factor VIII inhibitors
Roche has received clearance from the Food and Drug Administration for Hemlibra (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors.
Hemlibra is now the only prophylactic treatment for people with hemophilia A with and without factor VIII inhibitors that can be administered subcutaneously, under the skin, and at multiple dosing options — once weekly, every two weeks or every four weeks — the company said.
“Many preventative treatment options for people with hemophilia 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,”Michael Callaghan, Children’s Hospital of Michigan and Detroit hematologist said in a statement. “The approval of Hemlibra is an important advancement for the entire hemophilia 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.”
“Today’s approval of Hemlibra reflects our commitment to groundbreaking science and the development of medicines with the potential to redefine the standard of care,” Roche’s chief medical officer and head of global product development Sandra Horning said in a statement. “Hemlibra is now the only FDA-approved medicine for people with hemophilia A with and without factor VIII inhibitors, based on the efficacy and safety profile demonstrated across four pivotal studies. We want to thank the hemophilia community for their partnership in helping us bring this new option to everyone living with hemophilia A.”
Hemlibra was granted Breakthrough Therapy Designation by the FDA for hemophilia A without factor VIII inhibitors. It also was granted Priority Review, a designation given to medicines that the FDA has determined to have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease.
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