Gamifant (emapalumab-lzsg) / SOBI - LARVOL DELTA

Anti-CD123 CAR-T therapy combined with autologous SCT and venetoclax maintenance in refractory BPDCN ineligible for allogeneic transplantation: a case report and review of the literature. (PubMed, Front Immunol) - "The clinical course was complicated by Grade 3 cytokine release syndrome and suspected immune effector cell-associated HLH-like syndrome (IEC-HS), which were successfully managed with glucocorticoid and emapalumab. The patient achieved sustained MRD-negative CR with a disease-free survival exceeding 13 months. This multimodal paradigm-combining intensive cytoreduction, targeted immunotherapy with marrow rescue, and molecular maintenance-provides a feasible and potentially curative alternative for BPDCN patients in the "no-donor" setting."

Journal • Review • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Oncology • Transplantation • CD123 • IL3RA

Efficacy and Safety of Emapalumab in Adults with Macrophage Activation Syndrome (MAS) Secondary to Still's Disease: Results from a Pooled Analysis of Two Prospective Trials (ACR Convergence 2026) - No abstract available

Retrospective data • Immunology

Early-Onset Hemophagocytic Lymphohistiocytosis and Inflammatory Neurotoxicity Prior to Post-transplant Cyclophosphamide: A Report of Two Cases. (PubMed, Cureus) - "Case 1 developed severe cytokine release syndrome with shock, respiratory failure, renal failure, hyperferritinemia (22,771 ng/mL), hypertriglyceridemia (1,024 mg/dL), and multiorgan dysfunction, consistent with secondary hemophagocytic lymphohistiocytosis; he improved rapidly after receiving emapalumab. Case 2 developed fever and abrupt encephalopathy on day +2 with negative infectious and neurologic evaluation and rapidly improved after tocilizumab, consistent with cytokine-mediated neurotoxicity resembling immune effector cell-associated neurotoxicity syndrome. These cases highlight diagnostic overlap with infection and the need for early immunosuppressive intervention."

Journal • CNS Disorders • Dyslipidemia • Hemophagocytic lymphohistiocytosis • Hypertriglyceridemia • Immunology • Infectious Disease • Inflammation • Rare Diseases • Renal Disease • Respiratory Diseases • Transplantation

Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study (clinicaltrials.gov) - P2 | N=5 | Recruiting | Sponsor: University of Miami | Not yet recruiting ➔ Recruiting

Enrollment open • Dermatomyositis • Immunology • Interstitial Lung Disease • Myositis • Pulmonary Disease • Respiratory Diseases • Rheumatology • IFIH1

Randomized Stepped Wedge Study of Emapalumab in APECED Enteritis (clinicaltrials.gov) - P2 | N=30 | Recruiting | Sponsor: National Institute of Allergy and Infectious Diseases (NIAID) | N=10 ➔ 30

Enrollment change • Candidiasis • Gastrointestinal Disorder • Immunology

Living Donor Liver Transplantation as Salvage Therapy for Steroid-Refractory Chronic Hepatic GVHD Following Allogeneic Hematopoietic Stem Cell Transplantation. (PubMed, Pediatr Transplant) - "LDLT may represent a viable option in select pediatric patients with irreversible chronic hepatic GVHD. This case underscores the importance of early multidisciplinary planning and highlights the absence of liver-specific biomarkers or targeted therapies to guide management. Dedicated research into preventive and regenerative strategies is warranted."

Journal • Aplastic Anemia • Bone Marrow Transplantation • Cholestasis • Epstein-Barr Virus Infections • Graft versus Host Disease • Hematological Disorders • Hepatology • Immunology • Pediatrics • Transplantation

A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia (clinicaltrials.gov) - P2 | N=32 | Recruiting | Sponsor: M.D. Anderson Cancer Center | Trial primary completion date: Aug 2026 ➔ Aug 2028

Trial primary completion date • Hematological Disorders

SHIELD: Severe Hepatitis Intervention With Emapalumab for Liver Disease (clinicaltrials.gov) - P2 | N=28 | Not yet recruiting | Sponsor: Children's Healthcare of Atlanta

New P2 trial • Hepatology • Inflammation • Liver Failure • Pediatrics

Emapalumab for the Treatment of Peri-Graft Hemophagocytic Lymphohistiocytosis Reactivation in a Patient With Familial Hemophagocytic Lymphohistiocytosis Type 3. (PubMed, Pediatr Blood Cancer) - No abstract available

Journal • Hemophagocytic lymphohistiocytosis • Immunology • Rare Diseases

Current outlook on the use of biological agents to improve outcomes in adult secondary hemophagocytic lymphohistiocytosis. (PubMed, Expert Opin Biol Ther) - "Apart from treating promptly the underlying trigger conditions, earlier incorporation of targeted anti-cytokine therapy such as anakinra is strongly favored these days. The cytotoxic etoposide shall be chiefly reserved for malignancy-associated HLH, including B-cell lymphomas, and refractory disease. Rituximab has led to superior outcomes when utilized to clear EBV-infected B-cell reservoirs...The anti-IFN-γ monoclonal antibody emapalumab has been approved for refractory HLH cases. The JAK 1/2 inhibitor ruxolitinib may prove useful in this space as well. The therapeutic arsenal is likely to evolve further in the direction of agents addressing specific pathophysiologic steps in asHLH. Targeting effector cytokines, their receptors, inflammasome pathways are promising future directions in asHLH therapeutics."

Journal • Review • B Cell Lymphoma • Epstein-Barr Virus Infections • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Pediatrics • Rare Diseases • IFNG

Chimeric antigen receptor-T cell therapy-induced cardiotoxicity: pathophysiological mechanisms and pharmacological intervention strategies. (PubMed, Pharmacol Res) - "We have further summarized clinical pharmacological interventions for such cardiotoxicity, including interleukin (IL)-6 receptor antagonists (tocilizumab), interferon-γ inhibitors (emapalumab), and Janus kinase inhibitors (ruxolitinib). These findings provide novel directions and drug candidates for the treatment of CAR-T cell therapy-induced myocardial toxicity. This review provides a comprehensive theoretical basis for optimizing cardiovascular safety management and developing novel targeted interventions for CAR-T cell therapy."

Journal • Review • Cardiovascular • Hematological Disorders • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Hypotension • Immunology • Myocardial Ischemia • Oncology • Rare Diseases • Solid Tumor • IFNG • IL1B • IL6 • TNFA

The Perilous Journey of Patients With Primary CNS-HLH to Bone Marrow Transplant: A Case Report. (PubMed, Case Rep Hematol) - "Both patients received HLH-directed therapy (dexamethasone, etoposide, and emapalumab) plus intensive supportive care. Despite advances in management strategies, timely recognition, aggressive treatment, and access to HSCT remain essential to survival. Broader clinical experience and collaborative studies are needed to optimize care strategies for CNS-HLH."

Journal • Bone Marrow Transplantation • CNS Disorders • Epilepsy • Hematological Disorders • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Immunology • Oncology • Rare Diseases • Respiratory Syncytial Virus Infections • Transplantation • PRF1

Long-Term Treatment With Emapalumab in an Adult Patient With Refractory Hemophagocytic Lymphohistiocytosis and Systemic Lupus Erythematosus: A Case Report. (PubMed, Case Rep Hematol) - "This case report highlights the potential of emapalumab as an effective therapeutic option for the treatment of relapsed/refractory HLH in adults, including in SLE- and malignancy-associated disease not responding to conventional therapy. It supports its role in achieving rapid disease control, enabling prolonged remission with extended use, and allowing successful retreatment in recurrent HLH, including in high-risk settings with poor outcomes such as malignancy-associated HLH."

Journal • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Myelodysplastic Syndrome • Oncology • Pain • Rare Diseases • Systemic Lupus Erythematosus • IFNG

Pathogenic JAK1 gain-of-function variant contributing to rapid progression of Systemic EBV+ T-cell Lymphoma of Childhood (ISTH 2026) - "Six months prior, she presented with HLH due to primary EBV infection that responded to emapalumab and was maintained on ruxolitinib. PET scan on initial presentation. Page 3 DOI*10.1016/j.rpth.2026.106591"

Clinical • Epstein-Barr Virus Infections • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Hepatology • Immunology • Infectious Disease • Liver Failure • Lymphoma • Rare Diseases • T Cell Non-Hodgkin Lymphoma • IFNG • JAK1

Targeting the cytokine storm: new therapies in the treatment of paediatric haemophagocytic lymphohistiocytosis. (PubMed, Expert Rev Clin Immunol) - "Emapalumab, alemtuzumab and ruxolitinib show promising results, however widespread use is limited by small, non-randomized trials in a heterogenous population. Further international collaborative efforts to develop clinical trials to put conventional chemotherapy treatments head-to-head with these newer therapies is critical for advancement of these agents."

Journal • Review • Bone Marrow Transplantation • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Oncology • Pediatrics • Rare Diseases • Transplantation • IFNG

Emapalumab plus conventional therapy with or without ruxolitinib for pediatric hemophagocytic lymphohistiocytosis: a single center retrospective study. (PubMed, Immunol Res) - "In this small-sample retrospective study, emapalumab showed a more pronounced trend toward alleviating fever, hypofibrinogenemia, and neutropenia than toward improving liver function among pediatric HLH patients. Adding ruxolitinib to the combination of emapalumab and conventional therapy did not demonstrate a clear additional clinical benefit, particularly in the EBV-associated HLH and relapsed/refractory subgroups. These findings are constrained by the study design and sample size and necessitate validation in larger, prospective studies."

Journal • Retrospective data • Hematological Disorders • Hemophagocytic lymphohistiocytosis • Immunology • Neutropenia • Pediatrics • Rare Diseases • IFNG

Modified treatment protocol for pediatric systemic lupus erythematosus-associated hemophagocytic lymphohistiocytosis with central nervous system involvement: a case report. (PubMed, Front Immunol) - "Subsequently, second-line salvage therapy, consisting of emapalumab in combination with teniposide and dexamethasone, was initiated to account for the CNS involvement. The patient achieved sustained, complete clinical response following use of this modified therapeutic approach. Our findings suggest that this combination regimen may offer a potentially feasible therapeutic alternative for managing patients with SLE-associated HLH accompanied by CNS involvement; however, given the single-case nature of this report, further validation in larger cohorts is needed."

Journal • Hemophagocytic lymphohistiocytosis • Immunology • Inflammatory Arthritis • Lupus • Pediatrics • Rare Diseases • Systemic Lupus Erythematosus

Emapalumab and eltrombopag rescue for prolonged immune effector cell-associated hemophagocytic syndrome after CAR T-cell therapy in multiple myeloma. (PubMed, Haematologica) - "Not available."

Journal • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Multiple Myeloma • Oncology

Retrospective study on allogeneic hematopoietic stem cell transplantation for the treatment of Hemophagocytic lymphohistiocytosis. (PubMed, Transpl Immunol) - "Allo-HSCT achieved high engraftment rates and acceptable a safety profile across different HLH subtypes. Achieving at least partial remission (in an inactive state) before transplantation significantly improves survival. For critically ill patients with an extremely high inflammatory burden, emapalumab serves as an effective salvage strategy for bridging transplantation. Furthermore, the cellular tracing of EBV-R using advanced sorting technologies is of great clinical value for differentiating simple viremia from early tumor relapse."

Journal • Retrospective data • Bone Marrow Transplantation • Epstein-Barr Virus Infections • Hemophagocytic lymphohistiocytosis • Immunology • Oncology • Rare Diseases • Transplantation

EMAPALUMAB IN TREATMENT-EXPERIENCED PRIMARY HLH: SAFETY, EFFICACY, AND TRANSPLANT OUTCOMES FROM THE OPEN-LABEL, SINGLE-ARM POST-AUTHORIZATION STUDY SOBI.EMAPALUMAB-104 IN CHINESE PATIENTS (EHA 2026) - P4 | "Treatment-experienced pHLH patients received emapalumab twice weekly (first dose 1 mg/kg; stepwise increases up to 10 mg/kg were permitted) on background glucocorticoid (GC; ≥5 mg/m 2 /day, dexamethasone equivalent, tapered or increased per investigator's judgement). These outcomes are broadly consistent with the pivotal NI-0501-04 primary analysis (Locatelli et al, 2020). They also support emapalumab as a well-tolerated and effective bridge to HSCT in this critically ill population of treatment- experienced Chinese patients with pHLH."

Clinical • Bone Marrow Transplantation • Hematological Disorders • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Pneumonia • Rare Diseases • Respiratory Diseases • Respiratory Syncytial Virus Infections • Septic Shock • Transplantation

IMMUNE EFFECTOR CELL–ASSOCIATED HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS FOLLOWING CAR T-CELL THERAPY: RESULTS OF A SINGLE-CENTER REAL-WORLD ANALYSIS (EHA 2026) - "CAR-T-cell products infused were ciltacabtagene autoleucel (cilta-cel) (4/14)), idecabtagene vicleucel (3/14), axicabtagene ciloleucel (axi-cel) (3/14), brexucabtagene autoleucel (2/14), tisagenlecleucel (1/14) and lisocabtagen maraleucel (1/14)...Treatment consisted primarily of corticosteroids (12/14) and anakinra (12/14). Refractory pts received intravenous immunoglobulin (5/14), tocilizumab (3/14), siltuximab (1/14), ruxolitinib (1/14), emapalumab (1/14), or etoposide (1/14)...Despite intensive immunosuppressive therapy, outcomes remain poor. Early biomarker-driven identification and multicenter studies are needed to improve diagnosis and management of IEC-HLH pts."

CAR T-Cell Therapy • Clinical • IO biomarker • Real-world • Real-world evidence • Burkitt Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Hepatology • Immunology • Infectious Disease • Influenza • Liver Failure • Lymphoma • Mantle Cell Lymphoma • Multiple Myeloma • Neutropenia • Rare Diseases • Respiratory Diseases • Septic Shock • Thrombocytopenia

TREATED WITH EMAPALUMAB AS A BRIDGE TO HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR REFRACTORY/RECURRENT EPSTEIN-BARR VIRUS-ASSOCIATED HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (EHA 2026) - "All patients had received prior therapy with ruxolitinib and etoposide-containing regimens. Patients who are able to receive sequential allo- HSCT achieve significant survival benefit. Emapalumab may act as a bridge to allo-HSCT for these patients."

Bone Marrow Transplantation • Epstein-Barr Virus Infections • Hematological Disorders • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Inflammation • Rare Diseases • Respiratory Diseases • IFNG

TARGETING TNFΑ AND IFNG REVERSES CAR-T SECRETOME–INDUCED HUMAN HEMATOPOIETIC STEM CELL IMPAIRMENT (EHA 2026) - "TNF α and IFN γ are key mediators of CAR-T secretome–induced hematotoxicity; their blockade preserves HSPC phenotype and in vivo repopulating capacity without impairing CAR-T cytotoxicity. These findings support evaluation of clinically approved cytokine-neutralizing biologics, including etanercept (TNF α inhibitor) and emapalumab (anti-IFN γ monoclonal antibody), as potential strategies to prevent or mitigate CAR-T–associated prolonged cytopenias."

CAR T-Cell Therapy • Hematological Malignancies • IFNG • TNFA

Updated population pharmacokinetic (popPK) model for emapalumab using data from four prospective clinical trials in patients with hemophagocytic lymphohistiocytosis/macrophage activation syndrome (MAS) (EULAR 2026) - P2, P2/3, P3 | "This resulted in a newly added covariate effect for patients with MAS comprising a 78% larger volume of the peripheral compartment, although this effect did not translate into differences in emapalumab exposure. Overall, patients with MAS appeared to have higher emapalumab exposure compared with patients with pHLH, although these differences could be attributed to the differences in age and body weight of participants in the respective studies."

Clinical • PK/PD data • Hemophagocytic lymphohistiocytosis • Immunology • Inflammatory Arthritis • Lupus • Rare Diseases • Systemic Lupus Erythematosus • IFNG

Updated exposure–safety analysis of emapalumab in patients with macrophage activation syndrome (MAS) (EULAR 2026) - P2, P3 | "Conclusions Increasing emapalumab exposure was not associated with an increased risk of moderate or severe events, serious, nor infection TEAEs, confirming that repeated administration and/or increasing doses of emapalumab are well tolerated by patients with MAS in Still’s disease. The correlation between high emapalumab exposure (AUC) and lower number of infections may be considered a beneficial effect of emapalumab treatment that improved disease control, allowing tapering of generalized immunosuppression (e.g. glucocorticoids)."

Clinical • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Inflammatory Arthritis • Rare Diseases • IFNG