Earlier today, the U.S. Food and Drug Administration (FDA) approved Genentech’s Hemlibra (emicizumab-kxwh) for patients with hemophilia A who have developed Factor VIII inhibiting antibodies.
The newly-approved therapy is intended to reduce or completely prevent the frequency of bleeding episodes in adult and pediatric patients.
Hemophilia A is a rare genetic condition in which the blood of a patient is unable to properly form clots due to missing or reduced levels of Factor VIII. The disorder is characterized by symptoms including recurrent painful bleeding episodes, some of which can be life-threatening. Hemlibra is the first new medicine in nearly 20 years for this patient population, most of whom are at risk for long-term joint damage and disability due to bleeding issues. Before today’s approval, the current standard of care included the previously regulated Advate and Kogenate, both used prophylactically.
“Reducing the frequency or preventing bleeding episodes is an important part of disease management for patients with hemophilia. Today’s approval provides a new preventative treatment that has been shown to significantly reduce the number of bleeding episodes in patients with hemophilia A with Factor VIII inhibitors,” said Richard Pazdur, M.D., acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and director of the FDA’s Oncology Center of Excellence in a press release
. “In addition, patients treated with Hemlibra reported an improvement in their physical functioning.”
Results from 2 of the largest piviotal clinical trials for people with hemophilia A with inhibitors were the impetus behind the approval. The first of which enrolled 109 male patients aged 12 years and older, with the randomized portion comparing Hemlibra to no prophylactic treatment in 53 patients who were treated with on-demand therapy with a bypassing agent prior to their enrollment. Patients taking Hemlibra experienced an 87% reduction in the rate of bleeds that necessitated treatment compared to those who did not receive the therapy. Data from the Phase 3 HAVEN 1 trial that assessed the efficacy, safety and pharmacokinetics of once-weekly subcutaneous emicizumab were published in the New England Journal of Medicine
The same results were found in the second trial which evaluated a single arm of 23 males under the age of 12. 87% of patients taking Hemlibra did not experience a bleeding episode that required treatment.
Common side effects of Hemlibra include reactions at injection sites, headache and joint pain.
, the FDA accepted Genentech’s Biologics License Application (BLA) and granted Priority Review for emicizumab. The company was also previously granted Breakthrough Therapy Designation and Orphan Drug Designation from the FDA.
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