In a prospective, noncontrolled, open label, multinational, multi-centre Phase III study (GENA-03) efficacy and safety of Nuwiq® were assessed in a pediatric study that enrolled 59 children aged 2 to 12 years. Fifty-seven (57) of these patients had at least 50 EDs. All patients had at least 50 previous EDs to a FVIII concentrate. The mean prophylactic dose was 38.9 IU/kg, administrated every other day or 3 times per week. A total of 45.8% of the patients did not experience any bleeding while receiving Nuwiq® prophylaxis. The annualized median (range) rate of all bleeding episodes (BEs) was 1.9 (0-20.7), with spontaneous BE rates of 0 (0-13.8) and traumatic BE rates of 1.57 (0-18.6). Prophylaxis with Nuwiq® was assessed to be excellent or good for spontaneous BEs in 96.6% of the patients, for traumatic in 98.3% and for all BEs 91.5% of the patients. Efficacy of Nuwiq® in the treatment of breakthrough bleeding was rated excellent or good for 82.4% of BEs. One or two infusions were sufficient to treat 81.3% of BEs and the median (range) number of infusions required to stop a BE was 1.0 (range 1–22). The mean dose for breakthrough bleeding treatment was 45.1 IU/kg.

Design: Prospective, non-controlled, open-label, multinational, multi-centre, Phase III study

Dosing: PK and IVR: 50 IU/kg, in comparison to previously given FVIII product (in 50% of patients)

Prophylactic treatment: 30–40 IU FVIII/kg every other day or 3 times weekly. Two dose escalations of +5 IU/kg each allowed in case of an inadequate response (>2 spontaneous BEs during 1 month)

Treatment of BEs:
Minor BEs:
20–30 IU FVIII/kg every 12–24 h until BE resolution
Moderate to major BEs:
30–40 IU FVIII/kg every 12–24 h until BE resolution
Major to life threatening BEs:
Initial dose of 50–60 IU FVIII/kg and then a dose of 20–25 IU FVIII/kg every 8–12 h until BE resolution

Duration: >50 EDs and >6 months

Participants: 59 male PTPs with severe hemophilia A, age 6.1 years (mean), (range of 2-12)