New interim data from the phase 3 HAVEN 6 trial of emicizumab (Hemlibra®) as prophylaxis has shown a safe and effective profile in patients with hemophilia A without factor VIII (FVIII) inhibitors.
The results will be presented on December 12, 2021, as part of the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition.
“Data at the [interim analysis] of the HAVEN 6 study indicate that emicizumab has a favorable safety profile and is efficacious in bleed prevention for persons with mild or moderate HA, which is in line with results from the HAVEN/STASEY studies,” the researchers said.
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A total of 71 patients were enrolled in the HAVEN 6 (NCT04158648) study and received treatment with emicizumab. During the trial, 46.5% of patients did not experience any bleeding events and 80.3% did not have bleeding events that required treatment.
The mean annualized bleed rate was 2.2 (95% CI, .31-7.53) for all bleeds and only 0.7 (95% CI, .01-5.13) for bleeds that required treatment.
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At least 1 adverse event (AE) was reported in 49 patients (69.0%), with headaches being the most common (14.1%). Most AEs (84.5%) were not related to treatment with emicizumab. Injection-site reactions were reported in 12.7% of patients and were related to treatment.
One patient had 2 AEs with a grade of 3 or higher and 4 participants reported 6 AEs, but none were deemed related to emicizumab. Antidrug antibodies were found in 2 participants but no clinical impact or impact on the pharmacokinetics of emicizumab was observed.
In the 47 participants who had been observed by week 25, a mean improvement of -1.77 (standard deviation, 2.94) was seen on the Hemophilia Joint Health Score 2.1. In the Comprehensive Assessment Tool of Challenges in Hemophilia, most domains were stable throughout the trial, with a small improvement in “social activity risk perception” seen among the adolescent patients.
Patients in the HAVEN 6 trial received loading doses of emicizumab of 3 mg/kg once a week for 4 weeks before moving into maintenance doses of 1.5 mg/kg once per week, 3 mg/kg every 2 weeks, or 6 mg/kg every 4 weeks.
Of the 71 patients analyzed in the study, 69 were male and had a median age of 23.0 years (range, 2-71). Twenty of the patients had mild hemophilia A while the other 51 had moderate, and 37 participants were receiving FVIII prophylaxis at baseline.
Reference
Negrier C, Mahlangu J, Lehle M, et al. Emicizumab prophylaxis in persons with mild or moderate hemophilia A: results from the interim analysis of the HAVEN 6 study. Oral presentation presented at: 63rd American Society of Hematology (ASH) Annual Meeting & Exposition: December 12, 2021; Atlanta, Georgia.
