Gamifant (emapalumab-lzsg) / SOBI 
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 0 Diseases   4 Trials   4 Trials   250 News 


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  • ||||||||||  Gamifant (emapalumab) / SOBI, Thymoglobulin (anti-thymocyte globulin (rabbit)) / Sanofi
    EMAPALUMAB AS A BRIDGE TO TRANSPLANT IN A PATIENT WITH STAT1 GOF MUTATION (ePoster Area) -  Feb 11, 2023 - Abstract #EBMT2023EBMT_1811;    
    Active, not recruiting --> Completed 6-year-old female with a T-cell functional defect due to STAT1 GOF mutation with history of chronic mucocutaneous candidiasis, multi-drug resistant mycobacterium abscesses, and failure to thrive, received an unmodified bone marrow haplo-identical BMT from her mother with myeloablative conditioning (Thymoglobulin
  • ||||||||||  Gamifant (emapalumab) / SOBI
    Protocol for the two-cohort, open-label, single-arm EMERALD study of emapalumab, an anti-interferon gamma monoclonal antibody, in patients with macrophage activation syndrome in rheumatic diseases () -  Feb 4, 2023 - Abstract #BSR2023BSR_644;    
    P2, P3
    Interventional phase • Informed consent • Male and female patients aged 6 months to 80 years at diagnosis of MAS • Patients with a diagnosis of MAS having shown an inadequate response to high dose GCs as per local standard clinical practice• Primary haemophagocytic lymphohistiocytosis • Confirmed malignancy • Evidence of leishmania infection, clinically active mycobacteria (typical and atypical), histoplasma capsulatum, salmonella infections, or latent tuberculosis • History of hypersensitivity or allergy to any component of the study drug • A bacillus Calmette-Guerin vaccine within 12 weeks prior to screening • Live or attenuated live (other than bacillus Calmette-Guerin) vaccine within 4 weeks prior to Screening • Pregnancy or lactation • At time of emapalumab initiation, treatment with: - Canakinumab, Janus kinase inhibitors, tumour necrosis factor inhibitors, tocilizumab - Anakinra at a dose >4 mg/kg • Use of etoposide for MAS within 1 monthMAS diagnostic criteria• Febrile patient presenting with ferritin >684 ng/mL • And any two of: - Platelet count ≤181 x109/L - Aspartate aminotransferase levels >48 U/L - Triglycerides >156 mg/dL - Fibrinogen levels ≤360 mg/dL The primary endpoint of the study is protocol-defined CR at Week 8 after first emapalumab administration. The ongoing EMERALD study is designed to address the unmet need for new efficacious and safe therapies for the treatment of MAS.
  • ||||||||||  Gamifant (emapalumab) / SOBI
    A CASE REPORT OF SEVERE DENGUE CAUSING SECONDARY HEMOPHAGOCYTIC LYMPHOHISTIOCYTOSIS (HLH) AND MOSF () -  Jan 25, 2023 - Abstract #SCCM2023SCCM_1288;    
    Patient developed progressive MOSF requiring aggressive critical care support and Emapalumab treatment with eventual improvement...Mortality rate for MOSF in 4+ systems is 75-88%, let alone the high mortality of HLH and severe dengue. Despite her severity of disease requiring aggressive interventions, she achieved complete resolution with minimal long-term effects.
  • ||||||||||  Gamifant (emapalumab) / SOBI, Olumiant (baricitinib) / Incyte, Eli Lilly
    Journal:  Hemophagocytic lymphohistocytosis in trisomy 21: successful treatment with interferon inhibition. (Pubmed Central) -  Nov 20, 2022   
    Targeting therapy toward interferon signaling in both HLH and autoimmunity in trisomy 21 may have potential therapeutic benefits. Further investigation is needed to determine if trisomy 21 may predispose to the development of HLH given this common pathway.
  • ||||||||||  Review, Journal:  A Review of Current and Emerging Therapeutic Options for Hemophagocytic Lymphohistiocytosis. (Pubmed Central) -  Nov 10, 2022   
    Historically, the clinician's armamentarium for managing HLH was sparse, with etoposide-based protocols serving as the standard of care...Second- or subsequent-line options now include hematopoietic stem cell transplantation, emapalumab, alemtuzumab, anakinra, ruxolitinib, and tocilizumab...Unfortunately, many unanswered questions remain. Additional studies are required to optimize dosing, schedules, treatment sequences, and indications for novel treatment options.
  • ||||||||||  Campath (alemtuzumab) / Sanofi, Gamifant (emapalumab) / SOBI, Jakafi (ruxolitinib) / Novartis, Incyte
    ETOPOSIDE FOR PRIMARY HLH – BETTER THAN ITS REPUTATION. (Session Hall 02) -  Oct 19, 2022 - Abstract #ESID2022ESID_363;    
    Contemporary data are lacking, but are essential to put novel treatment approaches (emapalumab, alemtuzumab, ruxolitinib) into perspective...8 patients (4 FHL) only received IVIG/steroids/ cyclosporine A, while 59 received major HLH-directed drugs (first-line etoposide in 90%)...However, salvage therapies were used in 1/3. Importantly, early HSCT of asymptomatic siblings resulted in 100% survival, emphasizing the potential benefit of newborn screening.
  • ||||||||||  Gamifant (emapalumab) / SOBI
    Journal:  Histiocytic Disorders of Childhood. (Pubmed Central) -  Oct 8, 2022   
    Outcomes of treated children are poor, with approximately 60% survival. Emapalumab, which targets interferon-γ signaling, was recently approved for patients with recurrent or refractory HLH, and additional cytokine-directed therapies are under investigation.
  • ||||||||||  Gamifant (emapalumab) / SOBI
    Journal:  Hemophagocytic Lymphohistiocytosis. (Pubmed Central) -  Sep 14, 2022   
    Hematopoietic stem cell transplantation is a curative treatment in familial forms. Targeted therapy with emapalumab was also recently reported to be effective.
  • ||||||||||  Gamifant (emapalumab) / SOBI, Kineret (anakinra) / SOBI
    Journal:  Mutations at the C-terminus of CDC42 cause distinct hematopoietic and autoinflammatory disorders. (Pubmed Central) -  Jul 15, 2022   
    N=250 --> 3 | Trial completion date: Dec 2024 --> Apr 2022 | Suspended --> Terminated; Sponsor Decision related to Business Priorities, Assessment of Development Options Pathogenic variants at the CDC42 C-terminus differently impact protein stability, localization, and function, and cause different diseases, with p.R186C specifically associated with neonatal-onset pancytopenia and severe autoinflammation/hemophagocytic lymphohistiocytosis requiring emapalumab and bone marrow transplantation, and p.C188Y and p.∗192Cext∗24 causing anakinra-sensitive autoinflammation.
  • ||||||||||  Sylvant (siltuximab) / Jazz, Gamifant (emapalumab) / SOBI, Actemra IV (tocilizumab) / Roche, JW Pharma
    Journal, CAR T-Cell Therapy, Cytokine release syndrome:  Potential Role of IFNγ Inhibition in Refractory Cytokine Release Syndrome Associated with CAR T-cell Therapy. (Pubmed Central) -  May 6, 2022   
    To illustrate, we describe a patient with B-cell acute lymphoblastic leukemia who developed refractory grade 4 CRS following CD19-directed chimeric antigen receptor T-cell therapy, treated with tocilizumab, methylprednisolone, siltuximab, and the IFNγ inhibitor emapalumab, with complete remission from leukemia for 12 months. See related article by Bailey et al., p. 136 (15).
  • ||||||||||  Gamifant (emapalumab) / SOBI
    What's on the horizon for HLH? () -  Apr 20, 2022 - Abstract #ASPHO2022ASPHO_489;    
    Discuss the use and rationale of novel approaches to therapy, such as biologic agents (e.g., emapalumab, IL-18 Binding Protein) and small molecule inhibitors (e.g., JAK inhibitors) in the treatment of HLH. Illustrate key aspects (e.g., patient selection, timing, approach) of the application of novel cellular therapeutic approaches in the management of HLH such as reduced intensity allogeneic hematopoietic cell transplantation and preclinical data focusing on autologous gene modified hematopoietic cell transplantation in HLH.
  • ||||||||||  Gamifant (emapalumab) / SOBI
    LIVER FAILURE IN A NEWBORN WITH DOWN SYNDROME () -  Apr 20, 2022 - Abstract #ASPHO2022ASPHO_259;    
    This case emphasizes complexities in diagnosing TAM in the setting of pancytopenia and is the first report of this entity with the comorbid hyper-inflammatory syndrome of HLH. This novel association is in keeping with the association of other myelopoiesis abnormalities and HLH.
  • ||||||||||  measles vaccine / Generic mfg.
    A RARE CASE OF SECONDARY HLH DUE TO VACCINE-STRAIN MEASLES IN A YOUNG CHILD WITH HEPATOBLASTOMA () -  Apr 20, 2022 - Abstract #ASPHO2022ASPHO_43;    
    Although therearerareindividualreports of HLH secondary toprimarymeasles infection,to our knowledgethis is the first report of secondary HLH due to vaccine-strain measles[1].Uncontrolledreplicationof vaccine-strain measlesishighly unusualand can bedifficultto diagnose when presentation is atypical.This case highlights an exceptionallyrare complicationofimmunosuppressive therapy in a patient with recentlive vaccination.We hope greater awareness can lead to earlier recognition andimproved outcomes.1.LagousiT,KorovessiP,PanagouliE,TsagrisV,KostaridouS.ARare Case of Measles-Associated HemophagocyticLymphohistiocytosisin an Infant.Cureus. 2020; 12(5):e8246.
  • ||||||||||  Gamifant (emapalumab) / SOBI, Bevyxxa (betrixaban) / AstraZeneca, Turalio (pexidartinib) / Daiichi Sankyo
    What Can the US Learn from Regulatory Decisions and Health Technology Assessments of New Drugs in Other Countries? (In-person) -  Mar 8, 2022 - Abstract #ISPOR2022ISPOR_55;    
    The average US treatment cost of new FDA drug approvals receiving negative appraisals from international agencies was $150,817/year. CONCLUSIONS Review of drug approvals and HTA agency recommendations in other countries can provide evidence to support clinical decision-making of new drugs by US health systems and payers.
  • ||||||||||  Campath (alemtuzumab) / Sanofi
    Clinical, Journal:  Systemic and Nodular Hyperinflammation in a Patient with Refractory Familial Hemophagocytic Lymphohistiocytosis 2. (Pubmed Central) -  Feb 4, 2022   
    She had molecular and genetic confirmation of perforin deficiency and was started on dexamethasone and etoposide per HLH-94...The patient had a laboratory response to salvage therapy with alemtuzumab and emapalumab, but progressive neurologic decline led to withdrawal of care. This report highlights HLH foci manifest as pulmonary/renal nodules, demonstrates the utility of monitoring an array of HLH biomarkers, and suggests possible benefit of earlier salvage therapy.
  • ||||||||||  Gamifant (emapalumab) / SOBI
    Safety of Emapalumab in Pediatric Patients with Primary Hemophagocytic Lymphohistiocytosis (HLH): Relationship to Treatment Exposure (GWCC - Hall B5, Level 1) -  Nov 5, 2021 - Abstract #ASH2021ASH_3664;    
    P=N/A, P2/
    Current conventional therapy for HLH is based on immunochemotherapies, namely etoposide and glucocorticoids; this treatment, however, is associated with opportunistic infections and severe myelotoxicity...Emapalumab was initiated at a dose of 1 mg/kg administered intravenously every 3 days, on a Background of dexamethasone 5-10 mg/kg/day...Conclusion : In this study, the exposure-safety evaluation did not reveal any significant relationships between exposure to emapalumab and observed incidence rates of AEs, serious AEs, infections, or IRRs. These findings support the primary evidence of a favorable benefit-risk profile of emapalumab across the dose range used in this fragile patient population.