Immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients with severe hemophilia A: interim results from a phase 3, prospective, multicenter, open-label study

• Published in: Expert review of hematology Vol. 16; no. 10; p. 793
• Main Authors: Sidonio, Jr, Robert F, Thompson, Alexis A, Peyvandi, Flora, Stasyshyn, Oleksandra, Yeoh, Seoh Leng, Sosothikul, Darintr, Antmen, Ali Bulent, Maggiore, Caterina, Engl, Werner, Ewenstein, Bruce, Tangada, Srilatha
• Format: Journal Article
• Language: English
• Published: England 03-10-2023
• Subjects: Factor VIII - adverse effects; Hemophilia A - drug therapy; Hemorrhage - drug therapy; Humans; Prospective Studies; Antibodies; hemophilia A; inhibitors; factor VIII; prophylaxis; safety
• ISSN: 1747-4094
• DOI: 10.1080/17474086.2023.2247160

To determine the immunogenicity, safety, and efficacy of rurioctocog alfa pegol in previously untreated patients (PUPs) with severe hemophilia A (HA).
This prospective, phase 3 study (NCT02615691) was conducted in PUPs, or patients with ≤2 exposure days (EDs) prior to screening, aged <6 years with severe HA. The primary endpoint was incidence of factor VIII (FVIII) inhibitor development. This protocol-specified interim analysis was conducted after 50 patients had completed ≥50 EDs without developing FVIII inhibitors or had developed a confirmed inhibitor at any time.
Of the enrolled patients, 59/80 (73.8%) received ≥1 dose of rurioctocog alfa pegol; 54 received prophylaxis, and 35 on-demand treatment. Incidence of inhibitor development was 0.19 (10/52). Total annualized bleeding rate (95% CIs) was 3.2 (2.0-5.0) for patients receiving prophylaxis and 3.2 (1.6-6.3) for on-demand treatment. Hemostatic efficacy of most bleedings was rated as 'excellent' or 'good' after 24 hours (122/131 [93.1%]) and at resolution (161/170 [94.7%]). Five patients received ≥1 dose of rurioctocog alfa pegol for immune tolerance induction (ITI) and 1 patient was defined as having ITI success. Thirteen patients experienced 14 treatment-related adverse events, including 10 cases of FVIII inhibitor development.
This is the first prospective study of rurioctocog alfa pegol for the treatment of PUPs with severe HA.
This trial is registered at ClinicalTrials.gov (CT.gov identifier: NCT02615691).