CS/EP=chromogenic substrate assay per European Pharmacopoeia; CS/ADJ=chromogenic substrate assay/label adjusted to one-stage assay.

• LEOPOLD I: A four-part, multicentre, open-label trial examining the safety, efficacy and pharmacokinetics of Kovaltry^® in previously treated patients, adults and adolescents, with severe hemophilia A (<1%). Patients in Part B were given prophylaxis treatment of 20–50 IU/kg 2–3x/week for 12 months, with two different potency assignments (CS/EP and CS/ADJ for 6 months each).^1,3.
• LEOPOLD II: A Phase II/III, open-label, randomized, multicentre trial examining the safety and efficacy of Kovaltry^® in previously treated patients, adults and adolescents, with severe hemophilia A (<1%). Patients were randomized to be treated with the on-demand or prophylaxis regimen (low dose [20–30 IU/kg], low frequency [2x/week] or high dose [30–40 IU/kg], high frequency [3x/week]) for 12 months, with two different potency assignments (CS/EP and CS/ADJ for 6 months each).^1,4.
• LEOPOLD Kids (Part A): A two-part, Phase III, multicentre, open-label trial examining the safety, efficacy and pharmacokinetics of Kovaltry^® in previously treated patients, pediatrics <12 years, with severe hemophilia A (<1%). Patients were given prophylaxis treatment of 25–50 IU/kg at least 2x/week for treatment of breakthrough bleeds and prevention bleeds during surgical procedures.^1,5.
• Patient-years of exposure = International Units sold/annual dose per patient.
• Reporting period is January 27, 2016 to August 31, 2020.