Roche Holding AG said a phase 3 trial of Hemlibra showed the drug significantly controlled bleeding in certain patients with hemophilia A.
The phase 3 study, called Haven 4, evaluated 48 patients with hemophilia A with or without factor VIII inhibitors who were previously treated with either factor VIII or bypassing agents.
Results from the study showed Hemlibra, when given every four weeks as a preventive therapy, or prophylaxis, provided clinically meaningful control of bleeding. In the trial, 56.1% of patients experienced zero treated bleeds, and 90.2% experienced three or fewer treated bleeds. No serious side effects related to Hemlibra were seen in the study.
Factor VIII is an essential blood-clotting protein and people with hemophilia A either lack or do not have enough of it. A serious complication to treatment occurs when patients become resistant by developing antibodies, or inhibitors, which block the replacement factor VIII, making it difficult to control bleeding.
In April, the U.S. Food and Drug Administration granted its second breakthrough designation to Hemlibra for treating patients 12 years or older who suffer from hemophilia A and have not developed resistance to factor VIII replacement therapies.
Hemlibra was first granted the designation in September 2015, and was approved by the FDA in November 2017 for preventing or reducing the frequency of bleeding episodes in hemophilia A patients with factor VIII inhibitors.
Roche is banking on Hemlibra and other key products to offset the company's loss in sales in the U.S. due to biosimilar threats.