Omeros Corporation is set to unveil significant clinical findings on narsoplimab's effectiveness in treating transplant-associated thrombotic microangiopathy (TA-TMA) at the upcoming 2025 Tandem Meetings in Honolulu, Hawaii. The presentations will showcase real-world outcomes from the company's expanded access program, offering new hope for patients with this serious transplant complication.
Dr. Michelle Schoettler, Assistant Professor at Emory University School of Medicine, will deliver a podium presentation detailing overall survival data from 128 allogeneic transplant patients treated with narsoplimab. This comprehensive dataset represents one of the largest cohorts of TA-TMA patients treated under a compassionate use program.
Breakthrough in Refractory Cases
In a separate poster presentation, Dr. Piyatida Chumnumsiriwath from the University of California, Irvine, will present findings from a single-center study focusing on adult TA-TMA patients who received narsoplimab after experiencing treatment failure with eculizumab. This research particularly addresses the critical need for alternative treatments in refractory cases.
Mechanism of Action and Regulatory Status
Narsoplimab, also known as OMS721, represents a novel approach to treating TA-TMA through its targeting of mannan-binding lectin-associated serine protease-2 (MASP-2). This fully human monoclonal antibody selectively inhibits the lectin pathway while preserving the classical complement activation pathway, crucial for maintaining immune responses to infections.
The drug's development has gained significant regulatory support, with the FDA granting both breakthrough therapy and orphan drug designations for TA-TMA treatment. Additionally, the European Medicines Agency has designated narsoplimab as an orphan drug for hematopoietic stem-cell transplant treatment.
Regulatory Pathway and Future Plans
Omeros currently has a biologics license application (BLA) under FDA review for narsoplimab in TA-TMA treatment. The company plans to resubmit the BLA and subsequently file a European marketing authorisation application (MAA) in 2025, marking important steps toward potential market approval.
The upcoming presentations at the Tandem Meetings, which combine the expertise of the American Society for Transplantation and Cellular Therapy and the Center for International Blood and Marrow Transplant Research, will provide crucial real-world evidence supporting narsoplimab's potential role in addressing this serious transplant complication.
