PHASE III PIVOTAL TRIALS
Phase III pivotal studies prove IDELVION has powerful efficacy with both 7- and 14-day dosing
ON 7- AND 14-DAY PROPHYLAXIS*
Long-lasting efficacy—evaluated up to 4 years in the phase III extension study2
Abbreviations: AjBR, annualized joint bleeding rate; AsBR, annualized spontaneous bleeding rate; IQR, interquartile range.
*Data based on matched-pairs design.
†IQR is the middle 50% of people in a clinical study.
Phase II/III study design: The safety and efficacy of IDELVION were evaluated in a prospective, open-label, multicenter clinical study of 63 male PTPs with hemophilia B (≤2% endogenous FIX activity) who received at least one infusion of IDELVION. Subjects were aged 12 to 61 years; including 7 adolescent subjects aged 12 to 17. Subjects were treated for up to 27 months.
Phase III extension study design: This multicenter, open-label phase III extension study investigated the long-term safety and efficacy of IDELVION for routine prophylaxis and on-demand treatment of bleeds. Hemophilia B PTPs (FIX ≤2%) (n=59) who participated in a phase III pivotal study or who underwent surgery with IDELVION and continued with prophylaxis were enrolled in the study. Enrolled subjects were males aged 13–63 (mean age: 36.1 years). Five subjects were between 12–17 years of age. During the study, subjects were treated for a median (range) of 36.8 (7, 49) months.
PROLONG-9FP surgical substudy design: This study enrolled male patients, ≤65 years old with severe hemophilia B (FIX activity ≤2%) requiring non-emergency surgery. A total of 30 surgeries have been conducted in 21 patients, including four surgeries in four pediatric patients.
“Before my surgery we discussed the right IDELVION dosage options. Doing what I could to control the outcome and improve results felt good.”Watch Tony’s full story
References: 1. Santagostino E, Martinowitz U, Lissitchkov T, et al. Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial. Blood. 2016;127(14):1761-1769. 2. Mancuso ME, Pan-Petesch B, Lissitchkov T, et al. Long-term safety and efficacy of rIX-FP prophylaxis with extended dosing intervals up to 21 days in adults/adolescents with hemophilia B. J Thromb Haemost. In press. 3. Curtin J, Santagostino E, Karim FA, Li Y, Seifert W, Négrier C. Simplifying surgery in haemophilia B: Low factor IX consumption and infrequent infusions in surgical procedures with rIX-FP. Thromb Res. 2020;188:85-89. 4. Gill JC, Roberts J, Li Y, Castaman G. Sustained high trough factor IX activity levels with continued use of rIX-FP in adult and paediatric patients with haemophilia B. Haemophilia. 2019. doi:10.1111/hae.13735.