PB0229 - DESIGNING THE RYPLAZIM® (PLASMINOGEN, HUMAN-tvmh) ARM OF ATHN TRANSENDS: SAFETY AND EFFECTIVENESS IN PEOPLE WITH PLASMINOGEN DEFICIENCY

Sunday, June 25, 2023

18:30 – 19:30 ET

Room: Exhibition

Presenting Author(s)

Lynn Malec, MD, MSc

Senior Medical Director
Versiti Blood Research Institute, Milwaukee, WI, USA; Division of Hematology & Oncology, Departments of Medicine and Pediatrics, Medical College of Wisconsin, Milwaukee, WI, USA
Milwaukee, Wisconsin, United States

Co-Author(s)

RB

Rebecca Bialas

Board Chair
Plasminogen Deficiency Foundation
Durham, North Carolina, United States
CO

Carrie O'Neill

Director, Research Programs
American Thrombosis and Hemostasis Network
Rochester, New York, United States
KT

Karen Thibaudeau

Director, External Biologics
Prometic Bioproduction
Laval, Quebec, Canada
EW

Elaine Warner

Medical Affairs Lead
Kedrion Biopharma
Fort Lee, New Jersey, United States
EW

Erin Weigel

Medical Science Liaison-Hematology
Kedrion Biopharma
Fort Lee, New Jersey, United States
Michael Recht, MD, PhD, MBA (he/him/his)

Chief Medical and Scientific Officer
National Hemophilia Foundation
New York, New York, United States

Background: Plasminogen deficiency type 1 (PLGD-1) is a rare, inherited, systemic disorder with an estimated incidence of 1-2 per million population due to homozygous/compound heterozygous variants in PLG, the gene coding for plasminogen (PLG). Clinical manifestations of PLGD-1 result from inadequately lysed extravascular fibrin leading to accumulation of pseudomembranes in tissues and organs resulting in impaired function, morbidity, decreased quality of life (QoL), and, at times, mortality. In 2021, plasma-derived plasminogen, human-tvmh (Ryplazim®) received regulatory approval for treatment of people with PLGD-1 (PwPLGD-1).

Aims: To design a registry assessing the safety and effectiveness of Ryplazim® in PwPLGD-1 within the framework of ATHN Transcends.

Methods: ATHN Transends (NCT04398628) assesses the safety and effectiveness of contemporary therapy for people with inherited bleeding and clotting disorders. The primary aim is to collect specific adverse events harmonized with the European Haemophilia Safety Surveillance. The study is constructed to allow the addition of product-specific modules. We have designed the Ryplazim® arm of ATHN Transcends to assess safety and effectiveness of Ryplazim® in PwPLGD-1. The study will last for a total of 7 years or when at least 100 participants have attained 100 exposure days. The study will be conducted in any PwPLGD-1 prescribed and using Ryplazim® receiving care through an ATHN-affiliate.

Results: ATHN Transcends has received central IRB approval and is currently being rolled out across participating American Thrombosis and Hemostasis Network (ATHN) affiliates in the United States. Enrollment in the Ryplazim® arm can begin as soon as a site opens ATHN Transcends to enrollment.

Conclusion(s): ATHN Transcends provides a real-world mechanism in which to collect safety and effectiveness data of contemporary therapies for PwPLGD-1. The Ryplazim® arm of ATHN Transcends will allow collection of Good Clinical Practice-grade data. Funding for the Ryplazim® arm is being provided by Kedrion Bipharma.