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

Complement-targeted therapies for transplant-associated thrombotic microangiopathy: recent advances. (PubMed, Front Pharmacol) - "Earlier non-complement-directed therapies, including calcineurin inhibitor modification, plasma exchange, defibrotide, and rituximab, showed limited and inconsistent benefit...Prospective and large cohort data support the clinical activity of eculizumab (C5 inhibitor), with meaningful improvements in survival and organ recovery. Ravulizumab (long-acting C5 inhibitor) has shown encouraging phase 3 results, and narsoplimab (MASP-2 inhibitor), which became the first approved treatment for TA-TMA, has demonstrated promising outcomes, including activity in some patients previously exposed to C5 inhibition. In addition, newer agents targeting C5, C3, or factor B are expanding the therapeutic horizon for TA-TMA, although efficacy data are still limited for some of these therapies. Overall, complement-targeted therapies represent a therapeutic advance in TA-TMA, and ongoing prospective studies will be crucial to define optimal agent selection, sequencing, and integration into..."

Journal • Review • Bone Marrow Transplantation • Hematological Disorders • Thrombocytopenia • Transplantation • Transplantation Associated Thrombotic Microangiopathy

The CHMP recommended against approval of Yartemlea (narsoplimab) for the treatment of adults and children from two years of age with haematopoietic stem cell transplant-associated thrombotic microangiopathy (Investing.com) - "Thrombotic microangiopathy is a serious and potentially life-threatening complication following HSCT, a procedure where the patient’s bone marrow is replaced by stem cells from a donor to form new bone marrow that produces healthy cells."

CHMP • Transplantation Associated Thrombotic Microangiopathy

Narsoplimab: First Approval. (PubMed, Drugs) - "Additionally, a Marketing Authorisation Application for narsoplimab for the treatment of TA-TMA is currently under regulatory review in the EU. This article summarises the milestones in the development of narsoplimab leading to this first approval for TA-TMA."

Journal • Pediatrics • Transplantation • Transplantation Associated Thrombotic Microangiopathy

Omeros Corporation…announced that the U.S. Centers for Medicare & Medicaid Services (CMS) has assigned a permanent Healthcare Common Procedure Coding System (HCPCS) J-code for YARTEMLEA (PharmiWeb) - "The J-code for YARTEMLEA (J1289) simplifies and streamlines billing and reimbursement for patients covered by U.S. government programs (e.g., Medicare) and commercial payers. The J-code will become effective July 1, 2026....YARTEMLEA is the first and only approved treatment for hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA), an often-fatal complication of hematopoietic stem cell transplantation."

US reimbursement • Transplantation Associated Thrombotic Microangiopathy

New Anticomplement Drugs in Nephrology - Mechanism and Indication. (PubMed, Kidney Blood Press Res) - "Anticomplement therapies represent a transformative advance in nephrology, offering targeted interventions that can improve outcomes for patients with complement-mediated kidney diseases. Their strategic use may reduce disease progression, manage inflammation, and mitigate organ damage, highlighting the potential of personalized treatment approaches in complement-driven renal disorders."

Journal • Review • ANCA Vasculitis • Atypical Hemolytic Uremic Syndrome • Complement-mediated Rare Disorders • Diabetic Nephropathy • Fibrosis • Focal Segmental Glomerulosclerosis • Glomerulonephritis • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Nephrology • Renal Disease • Systemic Lupus Erythematosus • Vasculitis

Safety Study of IgAN, LN, MN, & C3 Glomerulopathy Including Dense Deposit Disease Treated With OMS721 (clinicaltrials.gov) - P2 | N=31 | Terminated | Sponsor: Omeros Corporation | N=54 ➔ 31 | Unknown status ➔ Terminated; Study terminated by Sponsor

Enrollment change • Trial termination • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Renal Disease

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