Subcutaneous emicizumab (Hemlibra®) was found to serve as an effective treatment for people with hemophilia A across various dosing frequencies and sample populations in two HAVEN studies published in Haemophilia.
In the phase 3 HAVEN 3 study (ClinicalTrials.gov Identifier: NCT02847637), 152 patients were randomly assigned across 3 study arms and received either 3 mg/kg per week for 4 weeks followed by maintenance 1.5 mg/kg per week (Arm A), 3 mg/kg per week for 4 weeks followed by maintenance 3 mg/kg once every 2 weeks (Arm B), or no prophylaxis (Arm C). The patients were on prior episodic FVIII, had experienced 5 or more bleeds over the past 24 weeks, and did not have inhibitors.
The patients in both Arm A and Arm B tolerated emicizumab well and demonstrated a statistically significant 68% reduction in treated bleed rate (P <.0001), with more than 91% of patients experiencing 3 or fewer treated bleeds.
In the related phase 3 HAVEN 4 study (ClinicalTrials.gov Identifier: NCT03020160), the primary outcome measure was the efficacy and tolerability of subcutaneous maintenance emicizumab doses 4 mg/kg every 4 weeks to provide effective bleed control in people with hemophilia A both with and without inhibitors. Researchers enrolled 41 patients (5 had inhibitors) and administered 3 mg/kg emicizumab per week for 4 weeks followed by 6 mg/kg every 4 weeks.
Emicizumab was well tolerated by the patients, with 68.3% of the patients experiencing 0 treated bleeds and 95.1% of the patients experiencing 3 or fewer treated bleeds.
The most common adverse event in both studies was injection-site reaction. No patients had adverse events that led to emicizumab discontinuation or withdrawal from the study.
The authors of both studies concluded that emicizumab was a safe and efficacious treatment for people with hemophilia A both with and without inhibitors, and that it reduced bleed rates, demonstrating superiority over previous FVIII prophylaxis.
Disclosure: Multiple authors declare affiliations with the pharmaceutical industry. For a full list of disclosures please refer to the original study.
- Mahlangu J, Oldenburn J, Paz-Priel I, et al. Emicizumab prophylaxis administered once-weekly or every two weeks provides effective bleed prevention in persons with hemophilia A (PwHA) without inhibitors – Results from the phase III HAVEN 3 study [Abstract M-LBMED01-005 (854)]. Haemophilia. 2018;24(suppl. 5):210-212.
- Pipe S, Jimenez-Yuste V, Shapiro A, et al. Emicizumab subcutaneous dosing every 4 weeks is safe and efficacious in the control of bleeding in persons with hemophilia A (PwHA) with and without inhibitors: Results from Phase 3 HAVEN 4 study [Abstract M-LBMED01-006 (861)]. Haemophilia. 2018;24(suppl. 5):212-213.