"Emapalumab"

Emapalumab therapy for hemophagocytic lymphohistiocytosis prior to reduced-intensity transplantation improves chimerism. Hemophagocytic lymphohistiocytosis (HLH) is a hyperinflammatory disorder driven by interferon-gamma (IFN-γ). Emapalumab, an anti-IFN-γ antibody, is approved for the treatment of patients with primary HLH. Hematopoietic stem cell transplantation (HSCT) is required for cure of HLH. Reduced intensity conditioning (RIC) HSCT is associated with improved survival but higher incidences of mixed chimerism and secondary graft failure. To understand the potential impact of emapalumab on post-HSCT outcomes we conducted a retrospective study of pediatric patients with HLH receiving a first RIC-HSCT at our institution between 2014 and 2022 after treatment for HLH, with or without

Emapalumab-lzsg (Gamifant) - To treat primary hemophagocytic lymphohistiocytosis (HLH) Drug Approval Package: GAMIFANT (emapalumab-lzsg) * Skip to main page content * Skip to search * Skip to topics menu * Skip to common linksHHS U.S. Department of Health and Human Services U.S. Food and Drug Administration * Follow FDA * En EspañolSearch FDASubmit search * Popular Content * Home * Food * Drugs * Medical Devices * Radiation-Emitting Products * Vaccines, Blood & Biologics * Animal & Veterinary * Cosmetics * Tobacco Products * Home * Drugs * Drug Approvals and Databases * Drugs@FDADrug Approval Package: GAMIFANT (emapalumab-lzsg) * Share * Tweet * Linkedin * Pin it * More sharing options * Linkedin * Pin it * Email * Print Company: Novimmune S.A.Application Number

Efficacy and safety of emapalumab in macrophage activation syndrome. Macrophage activation syndrome (MAS) is a severe, life-threatening complication of systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still's disease (AOSD). The objective of this study was to confirm the adequacy of an emapalumab dosing regimen in relation to interferon-γ (IFNγ) activity by assessing efficacy and safety. The efficacy outcome was MAS remission by week 8, based on clinical and laboratory criteria. We studied emapalumab, a human anti-IFNγ antibody, administered with background glucocorticoids, in a prospective single-arm trial involving patients who had MAS secondary to sJIA or AOSD and had previously failed high-dose glucocorticoids, with or without anakinra and/or ciclosporin. The study foresaw

Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis. Primary hemophagocytic lymphohistiocytosis is a rare syndrome characterized by immune dysregulation and hyperinflammation. It typically manifests in infancy and is associated with high mortality. We investigated the efficacy and safety of emapalumab (a human anti-interferon-γ antibody), administered with dexamethasone year after the last dose of emapalumab, if transplantation was not performed. The planned 8-week treatment period could be shortened or extended if needed according to the timing of transplantation. The primary efficacy end point was the overall response, which was assessed in the previously treated patients according to objective clinical and laboratory criteria. At the cutoff date of July 20, 2017

Emapalumab An official website of the United States government Here's how you know Log inAccess keysNCBI HomepageMyNCBI HomepageMain ContentMain NavigationBookshelfSearch databaseBooksAll DatabasesAssemblyBiocollectionsBioProjectBioSampleBooksClinVarConserved DomainsdbGaPdbVarGeneGenomeGEO DataSetsGEO ProfilesGTRHomoloGeneIdentical Protein GroupsMedGenMeSHNLM and EffectsSummary of Use during LactationNo information is available on the clinical use of emapalumab during breastfeeding. Because emapalumab is a large protein molecule with a molecular weight of about 147,000 Da, the amount in milk is likely to be very low and absorption is unlikely because it is probably destroyed in the infant's gastrointestinal tract. Until more data become available, emapalumab should

Treatment with emapalumab in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated BCGitis. Emapalumab, a fully human anti-IFNγ monoclonal antibody, has been approved in the US as second-line treatment of primary hemophagocytic lymphohistiocytosis (HLH) patients and has shown promise in patients with graft failure (GF) requiring a second allogeneic hematopoietic stem cell transplantation (HSCT). The blockade of IFNγ activity may increase the risk of severe infections, including fatal mycobacteriosis. We report a case of secondary HLH-related GF in the context of HLA-haploidentical HSCT successfully treated with emapalumab in the presence of concomitant life-threatening infections, including disseminated tuberculosis (TB). A 4 years old

Emapalumab for the treatment of relapsed/refractory hemophagocytic lymphohistiocytosis. Emapalumab is a fully human immunoglobulin G1 monoclonal antibody directed against interferon-γ (IFN-γ), which in November 2018 received the first global approval for the treatment of pediatric and adult patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent, or progressive

Emapalumab Prevention of CAR-T Cell Associated Toxicities This research study involves assessing the impact of emapalumab as preventative management of CAR-T related cytokine release syndrome in participants with Non-Hodgkin's lymphoma (NHL). The research study involves the following study interventions: * Fludarabine and cyclophosphamide (Lymphodepleting Chemotherapy)* Axicabtagene Ciloleucel * Emapalumab This is a phase 2 multi-center, open label study that is evaluating the safety and efficacy of emapalumab in preventing toxicities associated with axicabtagene ciloleucel in subjects with second- or third-line large B-cell non-Hodgkin's lymphoma. A phase II clinical trials test the safety and effectiveness of an investigational drug to learn whether the drug works in treating a specific disease

A Study of Emapalumab for Pediatric Aplastic Anemia The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options. undefined

Emapalumab Efficacy in Children With Primary Hemophagocytic Lymphohistiocytosis The goal of this retrospective study is to assess efficacy and safety of emapalumab prescribed for the treatment in children with refractory from of pHLH. Primary hemophagocytic lymphohistiocytosis (pHLH) encompasses a group of genetically determined disorders, characterized by severe, often fulminant systemic provides support for the pivotal pathogenic role of interferon-γ (IFNg) in hemophagocytic lymphohistiocytosis. Emapalumab is a fully human IgG1 anti-interferon-γ monoclonal antibody that binds free and receptor-bound interferon-γ and inhibits its biologic activity. The data accumulated from the phase II/III clinical trial and reports of the small groups of patients demonstrate its efficacy in pHLL

A Pilot Study of Emapalumab for the Treatment of CAR T-Cell Therapy-Associated Prolonged Cytopenia To look at the safety and effectiveness of emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART. Primary Objectives:- To evaluate the efficacy of emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART.Secondary Objectives:- To evaluate safety and tolerability of emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART.Exploratory Objective:- To investigate biomarkers of response and resistance to emapalumab for the treatment of prolonged severe cytopenia in participants with LBCL who receive CART.

Emapalumab Treatment For Anticipated Clinical Benefit In Sepsis Driven By The Interferon-Gamma Endotype (The EMBRACE Trial) EMBRACE is a double-blind, randomized, placebo-controlled, phase IIa study that will be conducted in multiple Intensive Care Units (ICUs) and departments of Internal Medicine across Greece. It aims to investigate if treatment with emapalumab, a monoclonal antibody which blocks IFNγ, may improve the outcome of patients with sepsis driven by the IDS (endotype of IFNγ-driven sepsis) endotype. EMBRACE also aims to identify the best dosing regimen of emapalumab for the management of IDS. The EMBRACE trial aims to generate proof-of-concept if treatment with emapalumab, a monoclonal antibody which blocks IFNγ signaling, may improve the outcome of patients with sepsis driven

A Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of pHLH in Treatment Experienced Chinese Patients The goal of this post-authorization study is to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with pHLH. This is an open-label, multi center, single arm, post-authorization study aiming to describe safety and efficacy of emapalumab in treatment experienced Chinese patients with confirmed or suspected primary hemophagocytic lymphohistiocytosis (pHLH). The main objectives of the study are to collect safety and efficacy data on emapalumab in treatment experienced Chinese pHLH patients

Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE The purpose of this study is to assess the safety, tolerability and efficacy of emapalumab in children and adults with macrophage activation syndrome (sHLH/MAS) in Still's disease (including systemic juvenile idiopathic arthritis and adult onset Still's disease) or with sHLH/MAS

A Study to Evaluate Emapalumab in Japanese Healthy Volunteers. This is a randomized, placebo controlled and double-blinded study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD) and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects. undefined

Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT. This study is designed as an open-label, single arm, proof of concept study in order to determine the appropriate emapalumab dosing regimen neutralizing IFNγ in patients at risk of GF. Patients presenting CXCL9 levels above a defined threshold and other clinical criteria will be eligible to receive emapalumab.Both children and adults, with malignant and non-malignant underlying diseases, receiving allo-HSCT who are at high risk of GF as defined in the inclusion criteria will be included in the study. The main objective of the study is to determine the appropriate emapalumab dose regimen

Efficacy and Safety of Emapalumab and Anakinra in Reducing Hyperinflammation and Respiratory Distress in Patients With COVID-19 Infection. Hyper-inflammation, caused by a cytokine storm resulting from an exaggerated response of the immune system in the presence of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), is considered to represent one of the most important negative prognostic , parallel group, 3-arm, multicenter study to assess the efficacy and safety of Emapalumab or Anakinra, versus standard of care (SoC). Patients between 30 and 80 years will be eligible to participate in the study. The study is planned to consist of three groups, each comprising 18 patients. Treatment will be randomized to either Emapalumab+SoC, Anakinra+SoC or only SoC for two weeks. Follow-up visit

atezolizumab, avelumab, belantamab-mafodotin, cemiplimab, daratumumab, dostarlimab, durvalumab, emapalumab, ipilimumab, mosunetuzumab, obinutuzumab, polatuzumab-vedotin, ramucirumab, tremelimumab).For oral agentsFourteen oral agents were identified as high–moderate (abemaciclib, adagrasib, avapritinib, cabozantinib, enasidenib, fedratinib, lenvatinib, lomustine, midostaurin, mobocertinib, niraparib

in intravenously repeated doses; this approach resulted in satisfactory responses in several patients.62 67 Emapalumab, an anti-IFN-γ antibody, is the only targeted therapy that has been tested in a clinical trial (open-labelled single arm) in Still’s disease-related MAS.68 In patients with severe MAS and who had failed standard of care with high-dose GCs, treatment with emapalumab was associated with achievement of MAS remission in the great majority of the patients with a marked GC sparing effect and a reassuring safety profile. It is important to point out that emapalumab is not yet approved in Europe. Additionally, the potential interest of JAK inhibitors should be mentioned, since a few case reports reported efficacy of JAK1/JAK2 inhibitors—that is, ruxolitinib or baricitinib—in such patients69 70