Drugs / Emapalumab

Regulatory milestones approvals, filings & regulatory actions · 3 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Label expansion US (FDA) GAMIFANT 2025-06-27 fda.gov
Refused (EMA) EU (EMA) Gamifant 2021-01-07 europa.eu
Approved US (FDA) GAMIFANT 2018-11-20 fda.gov

Trials 20 · a red edge is where a trial was stopped

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 12 trials
Phase 1 NCT06996119 Jun 2026 → May 2027 expected acute myeloid leukemia, graft versus host disease, myelodysplastic syndrome City of Hope Medical Center Recruiting No outcome recorded
Phase 1 NCT01459562 Sep 2011 → Jan 2013 Swedish Orphan Biovitrum Completed No outcome recorded
Phase 210 trials
Phase 2 NCT07538336 Nov 2026 → Nov 2028 expected University of California, San Francisco Not yet recruiting No outcome recorded
Phase 2 NCT07486869 Jul 2026 → May 2027 expected amyopathic dermatomyositis, interstitial lung disease University of Miami Not yet recruiting No outcome recorded
Phase 2 NCT07567014 May 2026 → May 2028 expected large B-cell lymphoma, plasma cell myeloma NYU Langone Health Not yet recruiting No outcome recorded
Phase 2 NCT07202598 Nov 2025 → Mar 2031 expected autoimmune polyendocrine syndrome type 1 National Institute of Allergy and Infectious Diseases (NIAID) Recruiting No outcome recorded
Phase 2 NCT06550141 Sep 2024 → Aug 2026 overdue follicular lymphoma, high grade B-cell lymphoma, large B-cell lymphoma, primary mediastinal large B-cell lymphoma Massachusetts General Hospital Recruiting No outcome recorded
Phase 2 NCT06285825 Jul 2024 → Aug 2026 overdue M.D. Anderson Cancer Center Recruiting No outcome recorded
Phase 2 NCT06430788 May 2024 → May 2029 expected aplastic anemia, bone marrow failure syndrome Memorial Sloan Kettering Cancer Center Recruiting No outcome recorded
Phase 2 NCT05787574 Mar 2023 → Mar 2027 expected autoimmune lymphoproliferative syndrome Joseph Oved Recruiting No outcome recorded
Phase 2 NCT04731298 May 2021 → Apr 2022 disease related to hematopoietic stem cell transplant Swedish Orphan Biovitrum Terminated No outcome recorded
Phase 2 NCT03311854 Feb 2018 → May 2020 adult-onset Still disease, juvenile idiopathic arthritis, macrophage activation syndrome Swedish Orphan Biovitrum Completed No outcome recorded
Phase 2/34 trials
Phase 2/3 NCT06951971 Jun 2025 → Jun 2027 expected hemophagocytic syndrome The First Affiliated Hospital of Soochow University Not yet recruiting No outcome recorded
Phase 2/3 NCT03985423 Jun 2020 → Jun 2021 secondary hemophagocytic lymphohistiocytosis Swedish Orphan Biovitrum Terminated No outcome recorded
Phase 2/3 NCT02069899 Aug 2014 → May 2021 hemophagocytic syndrome Swedish Orphan Biovitrum Completed No outcome recorded
Phase 2/3 NCT01818492 Jul 2013 → Jan 2019 hereditary hemophagocytic lymphohistiocytosis Swedish Orphan Biovitrum Completed No outcome recorded
Phase 33 trials
Phase 3 NCT06038422 Sep 2023 → Sep 2024 hemophagocytic syndrome, refractory malignant neoplasm Beijing Friendship Hospital Unknown No outcome recorded
Phase 3 NCT05001737 Dec 2021 → Jun 2024 adult-onset Still disease, macrophage activation syndrome, systemic lupus erythematosus, systemic-onset juvenile idiopathic arthritis Swedish Orphan Biovitrum Completed No outcome recorded
Phase 3 NCT03312751 Feb 2019 → Aug 2021 hemophagocytic syndrome Swedish Orphan Biovitrum Completed No outcome recorded
Phase 41 trial
Phase 4 NCT05744063 Feb 2023 → Feb 2025 hereditary hemophagocytic lymphohistiocytosis Swedish Orphan Biovitrum Completed No outcome recorded
Also used as a comparator or background therapy in 4 trials
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 01 trial
Phase 0 NCT07244419 Comparator Jan 2026 → Jun 2029 expected disease related to hematopoietic stem cell transplant, transplant rejection Children's Hospital Medical Center, Cincinnati Recruiting No outcome recorded
Phase 11 trial
Phase 1 NCT04765553 Comparator Jan → Jun 2021 Swedish Orphan Biovitrum Completed No outcome recorded
Phase 2/31 trial
Phase 2/3 NCT04324021 Comparator Apr → Nov 2020 COVID-19 Swedish Orphan Biovitrum Terminated No outcome recorded
Phase 41 trial
Phase 4 NCT06504030 Background Aug 2024 → Aug 2026 overdue hemophagocytic syndrome The First Affiliated Hospital of Soochow University Not yet recruiting No outcome recorded

Evidence & citations 7 cited values

Every value below carries the sentence it was read from. 25 sources stand behind the page.

FieldValueCited text
Known as emapalumab “This is a randomized, placebo-controlled and double-blinded study to evaluate the PK, PD and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects,...” NCT04765553
20

“Refractory/recurrent hemophagocytic lymphohistiocytosis patients will be treated with GTP regimen (Emapalumab+Teniposide+Methylprednisolone)” NCT06038422

“1. Emapalumab, a monoclonal antibody that blocks interferon-gamma, a key driver of the overactive immune response seen in HLH.” NCT06951971

Emapalumab, if needed.” NCT06504030

NCT04731298

NCT07567014

NCT05787574

NCT04324021

NCT06550141

NCT06285825

NCT07486869

NCT06996119

NCT03312751

NCT07202598

NCT05001737

NCT07244419

NCT03985423

NCT07538336

NCT02069899

NCT03311854

NCT06430788

Known as Emapalumab-Lzsg ClinicalTrials.gov intervention name — accepted as the source's own label NCT05744063
Known as NI-0501 “The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic...” NCT03312751
2

NCT01818492

NCT01459562

Action Inhibit “a drug called emapalumab can be used to block the action of interferon-gamma” PMID 38662147 Apr 2024
Modality Monoclonal antibody “Anti-IFN Gamma Monoclonal AntibodyNCT01459562
Route Intravenous “Single Intravenous Doses of NI-0501” NCT01459562
Target IFNG “Emapalumab is a fully human monoclonal antibody that targets free and receptor-bound interferon-gamma (IFNγ)” PMID 38662147 Apr 2024