Yartemlea (narsoplimab-wuug) / Omeros, University of Leicester, Helion Biotech - LARVOL DELTA

Narsoplimab: Clinical Evidence in TA‑TMA Response Rate & Survival vs External Control (EBMT 2026) - "Supported by Omeros."

Clinical

Narsoplimab: Case Series (EBMT 2026) - "Supported by Omeros."

Clinical

Narsoplimab: Survival Data from Expanded Access Program (EAP) (EBMT 2026) - "Supported by Omeros."

NARSOPLIMAB IN CRITICALLY ILL PATIENTS WITH TA-TMA REQUIRING INTENSIVE CARE: A SINGLE CENTER STUDY (EBMT 2026) - "In this real-world cohort of critically ill allo-HSCT recipients, narsoplimab was feasible, well tolerated and associated with clinical responses despite the severity of illness. Although limited by small sample size, these findings support narsoplimab as a potentially effective therapeutic option for severe TA-TMA requiring intensive care. Larger multicentre studies are warranted."

Clinical • Acute Kidney Injury • Bone Marrow Transplantation • Critical care • Graft versus Host Disease • Immunology • Infectious Disease • Nephrology • Renal Disease • Respiratory Diseases • Transplantation Associated Thrombotic Microangiopathy

NARSOPLIMAB IN CRITICALLY ILL PATIENTS WITH TA-TMA REQUIRING INTENSIVE CARE: A SINGLE CENTER STUDY (EBMT 2026) - "In this real-world cohort of critically ill allo-HSCT recipients, narsoplimab was feasible, well tolerated and associated with clinical responses despite the severity of illness. Although limited by small sample size, these findings support narsoplimab as a potentially effective therapeutic option for severe TA-TMA requiring intensive care. Larger multicentre studies are warranted."

Clinical • Acute Kidney Injury • Bone Marrow Transplantation • Critical care • Graft versus Host Disease • Immunology • Infectious Disease • Nephrology • Renal Disease • Respiratory Diseases • Transplantation Associated Thrombotic Microangiopathy

I-SPY_COVID: I-SPY COVID-19 TRIAL: An Adaptive Platform Trial for Critically Ill Patients (clinicaltrials.gov) - P2 | N=1500 | Active, not recruiting | Sponsor: QuantumLeap Healthcare Collaborative | Recruiting ➔ Active, not recruiting

Enrollment closed • Infectious Disease • Novel Coronavirus Disease

REDUCED-TOXICITY MYELOABLATIVE TBI-BASED CONDITIONING FOR ≥2-MISMATCH PARTIALLY MATCHED RELATED DONOR HSCT WITH PTCY IN HIGH-RISK HEMATOLOGICAL MALIGNANCIES: SINGLE-CENTRE REAL-WORLD OUTCOMES FROM INDIA (EBMT 2026) - "Background: Partially matched related donor (PMRD) HSCT with post-transplant cyclophosphamide (PTCy) broadens access to allogeneic transplantation...Conditioning comprised fludarabine–TBI 8 Gy (predominantly ALL) and fludarabine–melphalan 75 mg/m²–TBI 4 Gy...Program specific modifications adapted from earlier clinical experience of program leads included (i) early UGIE for upper-GI symptoms enabling prompt diagnosis of predominantly upper-GI aGVHD, (ii) planned early immunosuppression taper/withdrawal (typically by ~day 60) to augment graft-versus-leukemia effect, (iii) universal prophylactic/pre-emptive granulocyte transfusions for all patients, and (iv) a resource-adapted CMV strategy with off-label leflunomide-based pre-emptive management for low-level CMV DNAemia before letermovir availability...CMV reactivation occurred in 5/10 and BK virus in 2/10; TA-TMA occurred in 1/10 (resolved with Rituximab , Narsoplimab) and VOD/SOS in 0/10... An RT-MAC TBI-based..."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Oncology

Narsoplimab-wuug. (PubMed, Am J Health Syst Pharm) - No abstract available

Journal

Diagnosing and Treating TA-TMA with Newly Approved Narsoplimab-wuug (TCT-ASTCT-CIBMTR 2026) - No abstract available

Transplantation Associated Thrombotic Microangiopathy

Use of narsoplimab for eculizumab-refractory adult transplant-associated thrombotic microangiopathy (TA-TMA). (PubMed, Ann Hematol) - "He was refractory to numerous treatments, including eculizumab, steroids, rituximab, and plasma exchange. After developing diffuse Alveolar hemorrphage and renal failure, he was initiated on Narsoplimab and later achieved a complete hematological response and became transfusion independent. This case highlights the importance of early recognition of TA-TMA and the need to switch therapy to other complement inhibitors if resistance to Eculizumab is noted."

Journal • Bone Marrow Transplantation • Chronic Kidney Disease • Hematological Disorders • Renal Disease • Thrombocytopenia • Transplantation • Transplantation Associated Thrombotic Microangiopathy

FDA nod to first-ever treatment for deadly post-transplant complication TA-TMA (Indian Pharma Post) - "'This approval is a long-awaited breakthrough in hematopoietic cell transplantation and TA-TMA care,' stated Miguel-Angel Perales...'Until now, we've lacked an effective TA-TMA therapy and relied largely on supportive measures such as modifying calcineurin inhibitors, which can significantly increase the risk of life-threatening graft-versus-host disease. Based on a compelling data package, narsoplimab delivers robust response rates and improved survival in TA-TMA, with a favorable benefit-risk profile and a safety profile consistent with that seen in patients undergoing hematopoietic stem cell transplantation. As the first and only drug approved for TA-TMA, narsoplimab is a practice-changing advance for patients facing this devastating complication.'"

Media quote • Regulatory

FDA Approves Omeros’ YARTEMLEA - First and Only Therapy Indicated for TA-TMA (Businesswire) - "'This approval is a long-awaited breakthrough in hematopoietic cell transplantation and TA-TMA care,' stated Miguel-Angel Perales, MD...'Until now, we’ve lacked an effective TA-TMA therapy and relied largely on supportive measures such as modifying calcineurin inhibitors, which can significantly increase the risk of life-threatening graft-versus-host disease. Based on a compelling data package, narsoplimab delivers robust response rates and improved survival in TA-TMA, with a favorable benefit-risk profile and a safety profile consistent with that seen in patients undergoing hematopoietic stem cell transplantation. As the first and only drug approved for TA-TMA, narsoplimab is a practice-changing advance for patients facing this devastating complication.'"

Media quote • Regulatory

FDA Approves Omeros’ YARTEMLEA – First and Only Therapy Indicated for TA-TMA (Businesswire) - "Approval of YARTEMLEA was based on results from a single-arm, open-label study in adults with TA-TMA (the TA-TMA Study; N=28), supported by additional data from an expanded access program (EAP; N=221 adult and pediatric patients). In the EAP, 19 patients (13 adult and 6 pediatric) had evaluable patient-level response data...Following FDA approval of YARTEMLEA, Omeros is finalizing preparations for its U.S. product launch planned for January 2026...A marketing authorization application for YARTEMLEA for the treatment of TA-TMA is currently under review by the European Medicines Agency with a decision expected in mid-2026."

EMA approval • FDA approval • Launch US • Hematological Disorders • Transplantation

Complement inhibition in post-transplant IgA-mediated autoimmune cytopenia: A pediatric case report (ASH 2025) - "He developed cellular rejection one-year post-transplant and was treated with cyclosporine and MMF. At 7 years post-transplantation, he developed acute cellular rejection with third-degree heart block requiring pulse steroids and ATG, with subsequent transition to tacrolimus/sirolimus...Modification of immunosuppression is particularly challenging in the post solid organ transplant setting and can risk organ rejection requiring close collaboration with primary transplant team.⁵⁻⁶ In IgA-driven diseases like IgA nephropathy, complement inhibitors (e.g., eculizumab, narsoplimab) are gaining traction.⁷ This case adds to emerging evidence that complement modulation is a viable treatment pathway for refractory IgA-mediated AIHA, especially in the post-transplant setting. Eculizumab with prednisone was effective and well-tolerated in this case of IgA-mediated, rituximab-refractory AIHA post-heart transplant. Complement inhibition may represent a novel and safe therapeutic..."

Case report • Clinical • Post-transplantation • Autoimmune Hemolytic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • IgA Nephropathy • Immunology • Pediatrics • Renal Disease • Solid Organ Transplantation • Thrombocytopenia • Transplantation

aHUS: Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome (clinicaltrials.gov) - P3 | N=6 | Terminated | Sponsor: Omeros Corporation | N=80 ➔ 6 | Unknown status ➔ Terminated; Sponsor terminated study

Enrollment change • Trial termination • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Hematological Disorders • Nephrology • HP

Characterization of a Long-Acting Anti-Human MASP-2 Antibody for the Treatment of Complement-Related Diseases. (PubMed, J Inflamm Res) - "Preclinical studies demonstrated that SHR-2010 exhibited superior pharmacokinetics and sustained lectin pathway inhibition compared to OMS721. When coupled with optimized trial design strategies, SHR-2010 could be a promising therapeutic candidate for lectin pathway-driven diseases, including IgAN."

Journal • Acute Kidney Injury • Bone Marrow Transplantation • Glomerulonephritis • IgA Nephropathy • Inflammation • Nephrology • Rare Diseases • Renal Disease • Transplantation

Transplant Associated Thrombotic Microangiopathy: Acomprehensive Review and Local Experience (ASH 2023) - "eculizumab, a humanized antibody against complement protein, can be highly effective in patients with TA-TMA...Other available treatment options include rituximab and defibrotide. Other therapeutic agents are under clinical trials such as ravulizumab (C5 inhibitor), nomacopan (C5 and leukotriene B4 inhibitor) and narsoplimab (mannan-binding lectin-associated serine protease-2 [MASP-2] inhibitor)...Thus far, the choice is to individualize therapy according to comorbidities, severity of clinical presentation and availability of the treatment options. ConclusionTA-TMA remains a significant clinical challenge for transplant physicians, and more research is needed to improve our understanding of its pathogenesis, diagnosis, and management, particularly in guiding the choice of therapy."

Review • Anemia • Bone Marrow Transplantation • Cardiovascular • Graft versus Host Disease • Hematological Disorders • Hypertension • Immunology • Infectious Disease • Thrombocytopenia • Transplantation

Clinical Safety and Efficacy of Narsoplimab in Pediatric and Adult Patients with Transplant-Associated Thrombotic Microangiopathy: A Real-World Experience (ASH 2023) - "Three of the four patients who failed to respond, eventually died with laboratory and clinical evidence of persisting TA-TMA. Conclusion In this study of high-risk TA-TMA patients, the inhibition of the lectin pathway of complement activation with narsoplimab was shown to be not only an effective but also a remarkably safe treatment option with no evidence of an increased risk of infectious complications in both children and adults."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Anemia • Bone Marrow Transplantation • Cardiovascular • Graft versus Host Disease • Hematological Disorders • Hypertension • Immunology • Infectious Disease • Pediatrics • Thrombocytopenia • Transplantation • HP

Management of a Complex Case of Transplant-Associated Thrombotic Microangiopathy in a Pediatric Patient with High-Risk Neuroblastoma (ASH 2024) - "Initial treatment included Eculizumab with minimal response. Treatment was changed to Narsoplimab (OMS721), and Defibrotide was added with minimal response...Defibrotide was discontinued, and further therapy with plasmapheresis/PLEX (Plasma Exchange) and Rituximab was added...The case underscores the necessity of a multidisciplinary approach to address TA-TMA and highlights the importance of adjusting therapy based on the response.ConclusionTA-TMA in patients with high-risk neuroblastoma is rare but is a serious complication of treatment, and the management is complex. This case demonstrates the need for a coordinated, personalized treatment strategy to optimize treatment outcomes."

Clinical • Anemia • Bone Marrow Transplantation • Cardiovascular • Hematological Disorders • Hypertension • Nephrology • Neuroblastoma • Oncology • Pediatrics • Renal Disease • Solid Tumor • Thrombocytopenia • Transplantation • HP

Survival in Adults with High Risk TA-TMA - A Comparative Analysis of Narsoplimab Versus Supportive Care. (PubMed, Blood Adv) - P, P2 | "In conclusion, in patients with high-risk TA-TMA, narsoplimab treatment significantly reduced mortality relative to a well-matched external control group who did not receive narsoplimab. These results support narsoplimab as a potential therapeutic option for TA-TMA."

Journal • Bone Marrow Transplantation • Transplantation

The FDA has assigned a PDUFA target action date of December 26, 2025, for its review of narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy/TA-TMA. (RTTNews)

PDUFA • Transplantation

Omeros Announces Publication Highlighting Survival Outcomes in TA-TMA Patients Treated with Narsoplimab Under Its Global Expanded Access Program (Yahoo Finance) - "Authored by an international panel of pediatric and adult transplant experts, the manuscript – titled 'Narsoplimab Results in Excellent Survival in Adults and Children with Hematopoietic Cell Transplant Associated Thrombotic Microangiopathy (TA-TMA)' – reports survival outcomes in patients treated with narsoplimab as first-line therapy and in those who failed prior treatments, including C5 inhibitors. Consistent with previous narsoplimab clinical studies, no safety signals of concern were observed."

Clinical data • Transplantation

Narsoplimab Results in Excellent Survival in Adults and Children With Hematopoietic Cell Transplant Associated Thrombotic Microangiopathy (TA-TMA). (PubMed, Am J Hematol) - "One-year overall survival (OS) in pediatric allogeneic recipients with HR TA-TMA who received narsoplimab as first-line therapy (n = 12) was 75.0% and 56.2% in those who received treatment as ≥ second-line (n = 25, 20 refractory to eculizumab). There were no concerning safety signals. In this real-world study enriched with patients with severe TA-TMA, survival was excellent in children and adults, supporting the use of narsoplimab."

Journal • Oncology • Pediatrics • Solid Tumor • Transplantation

FDA delays Omeros' 2nd attempt at transplant drug's approval by 3 months (FierceBiotech) - "Omeros had been given a deadline of Sept. 25 for the agency to reconsider whether to approve the anti-MASP-2 antibody narsoplimab for hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)...The FDA had requested further information in relation to Omeros’ application and, after receiving the biotech’s response, informed the company that the decision date has been pushed back to Dec. 26...The agency explained that 'assuming no major deficiencies are identified during its review, labeling discussions are planned to begin no later than Oct. 2025'..."

FDA event • PDUFA • Transplantation

Targeting the Roots of Kidney Disease: Systematic Review of the Therapies Targeting the Complement System. (PubMed, Medicina (Kaunas)) - "Meanwhile, avacopan, a C5a receptor antagonist, addresses ANCA-associated vasculitis (AAV) by mitigating inflammation and enabling reduced reliance on corticosteroids. Similarly, narsoplimab, which inhibits MASP-2, targets the lectin pathway implicated in conditions such as aHUS. Iptacopan, a factor B inhibitor, focuses on the alternative pathway and demonstrates efficacy in managing C3 glomerulopathy (C3G). A systematic review of complement-targeted therapies was conducted, analysing studies from 2013 to 2023 that address unmet medical needs in primary and secondary glomerular diseases. Our systematic review of complement-targeted therapies shows that these tailored and innovative treatments may specifically address unmet medical needs in primary and secondary glomerular diseases."

Journal • Review • ANCA Vasculitis • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • Inflammation • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Disease • Vasculitis