Helimbra – pro
The FDA initially approved emicizumab-kxwh in November 2017 for adults and children with hemophilia A with factor VIII inhibitors.
The latest approval is based on the pivotal phase 3 HAVEN 3 and HAVEN 4 clinical trials, part of the largest clinical program in hemophilia A. The HAVEN 3 study, which included 152 participants, evaluated emicizumab-kxwh prophylaxis administered every week or 2 weeks in patients aged 12 years or older with hemophilia A without factor VIII inhibitors. In the HAVEN 4 study, emicizumab-kxwh was administered every 4 weeks.
Data from the HAVEN 3 trial showed that administration of emicizumab-kxwh prophylaxis every week or every 2 weeks led to a 96% and 97% reduction in treated bleeds, respectively, compared with those who received no prophylaxis. Additionally, 55.6% of patients administered emicizumab-kxwh every week and 60% who received emicizumab-kxwh every 2 weeks experienced 0 bleeds.
Furthermore, once-weekly emicizumab-kxwh prophylaxis showed superior efficacy compared with prior factor VIII prophylaxis as demonstrated by a 68% reduction in treated bleeds in an intra-patient comparison.
In the HAVEN 4 study, which included 48 patients, emicizumab-kxwh prophylaxis showed clinically meaningful control of bleeding in adults and adolescents aged 12 years or older with hemophilia A without factor VIII inhibitors. According to the findings, 56.1% of patients experienced 0 treated bleeds and 90.2% experienced 3 or fewer treated bleeds.
HEMLIBRA is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors.. Phase 3 HAVEN 2 study’s results shows that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis (preventive treatment) leads to significant reductions in the number of bleeds among hemophilia A children younger than 12 with factor VIII inhibitors.
The results showed that Hemlibra administered on different schedules — once a week, either every two or four weeks — resulted in a clinically meaningful reduction of bleeds.
Guy Young et al. Ab 63,2 Emicizumab Prophylaxis Provides Flexible and Effective Bleed Control in Children with Hemophilia Α with Inhibitors: Results from the HAVEN 2 Study
ASH, Monday, December 3, 2018:
Helimbra, Prescribing Information 2019
Johnny Mahlangu, M.B., B.Ch., M.Med., Johannes Oldenburg, M.D., Ph.D., Ido Paz-Priel, M.D., Claude Negrier, M.D., Ph.D., Markus Niggli, Ph.D., M. Elisa Mancuso, M.D., Ph.D., Christophe Schmitt, Pharm.D., Victor Jiménez-Yuste, M.D., Ph.D., Christine Kempton, M.D., Christophe Dhalluin, Ph.D., Michael U. Callaghan, M.D., Willem Bujan, M.D., et al.Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors. N Engl J Med 2018; 379:811-822