Regulatory Decision Summary for Esperoct

Haemophilia A (congenital factor VIII [FVIII] deficiency) is a serious and potentially life-threatening bleeding disorder. Factor replacement therapy is the primary treatment for patients with haemophilia.

The benefits of Esperoct have been showed in routine prophylaxis, control of bleeding episodes, and perioperative management. The prophylactic effect of Esperoct was demonstrated with an annualized bleeding rate (ABR) of 1.95 in patients < 12 years of age with 60 IU/kg (50-75 IU/kg) twice weekly, and 1.18 in patients >12 years of age with 50 U/kg every 4 days. Of the 1,506 bleeds, 1,314 (87.3%) were rated "excellent" or "good" in their response to Esperoct. The hemostatic effect of Esperoct was rated as "excellent" or "good" in 43 of 45 major surgical procedures (95.6%). Note that the treatment success of the Q7D schedule (75 IU/kg every 7 days) for routine prophylaxis was not established.

The safety profile of Esperoct derived from the clinical trials is generally considered acceptable. The most frequently reported adverse reactions were rash (5.2%), injection site reaction (2.6%), erythema (1.9%), and pruritus (1.5%). One previously treated subject developed confirmed neutralizing antibodies to Factor VIII; the risk of development of inhibitory antibodies is considered an expected adverse event. No PEG was detected in brain tissues in the long-term repeat-dose toxicity studies with Esperoct. The potential risks of Esperoct have been addressed through the product monograph (PM) and the Risk Management Plan (RMP).

Based on the review of the submitted data, Esperoct has demonstrated safety and efficacy in adults and children with hemophilia A for the proposed three indications (routine prophylaxis to prevent or reduce the frequency of bleeding episodes, on-demand treatment and control of bleeding episodes, and perioperative management of bleeding). The benefit-risk profile of Esperoct in adults and children with hemophilia A is considered favorable.