ASH Clinical News Focus on Rare Diseases | Page 8

Designed with his future in mind Proven efficacy and safety in adolescents and adults with prophylaxis using as few as 2 infusions per week1 LEOPOLD I Trial1,2 Study description Dosing Multinational, open-label, prospective trial evaluating pharmacokinetics, efficacy, safety, and perioperative management of bleeding with KOVALTRY® Previously treated male patients (PTPs) aged 12 to 65 years with severe hemophilia A (<1% FVIII) (n=73) studied for 1 year Dosing regimens were determined by the investigators to meet individual patients’ needs 2x/week prophylaxis: 20-50 IU/kg (n=18) 3x/week prophylaxis: 20-50 IU/kg (n=44) Primary efficacy endpoint Annualized bleed rate (ABR) at 12 months (n=62 for efficacy analysis) After a single 50 IU/kg dose of KOVALTRY®, the demonstrated half-life [mean ± standard deviation (SD)] in 26 previously treated adolescent and adult patients was: Pharmacokinetics 12 to 17 years (n=5) Chromogenic Assay One-stage Assay 14.4 ± 5.5 hours 11.7 ± 1.1 hours ≥18 years (n=21) 14.2 ± 3.5 hours 14.3 ± 3.7 hours LEOPOLD=Long-Term Efficacy Open-Label Program in Severe Hemophilia A Disease. SELECTED IMPORTANT SAFETY INFORMATION Hypersensitivity reactions, including anaphylaxis, are possible with KOVALTRY®. Early signs of hypersensitivity reactions, which can progress to anaphylaxis, may include chest or throat tightness, dizziness, mild hypotension and nausea. Discontinue KOVALTRY® if symptoms occur and seek immediate emergency treatment. KOVALTRY® may contain trace amounts of mouse and hamster proteins. Patients treated with this product may develop hypersensitivity to these non-human mammalian proteins. Please see additional Important Safety Information on following pages. For additional important risk and use information, please see Brief Summary on following pages.