Plasminogen, human‐tvmh for the treatment of children and adults with plasminogen deficiency type 1

Abstract

AbstractAimAn open‐label phase 2/3 study of plasminogen, human‐tvmh administered intravenously in paediatric and adult subjects with type 1 plasminogen deficiency was conducted. Interim data was previously reported. The final data on 15 subjects who completed the study up to a maximum of 124 weeks are reported here.MethodsThe primary objectives were to evaluate efficacy of plasminogen replacement therapy on clinically evident or visible lesions during 48 weeks of dosing and to achieve an increase in trough plasminogen activity levels by at least an absolute 10% above baseline during 12 weeks of treatment.ResultsThe primary efficacy endpoint was achieved, as 100% of subjects (n = 11) with visible and assessable non‐visible lesions at baseline demonstrated ≥ 50% improvement after 48 weeks of study drug treatment with plasminogen, human‐tvmh. All subjects achieved the targeted ≥ 10% increase in trough plasminogen activity above baseline through Week 12. Plasminogen, human‐tvmh at a dose of 6.6 mg/kg administered every 2–5 days for 48 weeks and every 1–7 days for up to 124 weeks was well tolerated.ConclusionThis study provides additional evidence regarding the long‐term safety and clinical utility of replacement therapy with human plasminogen for the treatment of children and adults with type 1 plasminogen deficiency. Plasminogen, human‐tvmh received marketing approval on June 4, 2021. This trial was registered at www.clinicaltrials.gov as #NCT02690714.