Drugs / Emapalumab
Regulatory milestones approvals, filings & regulatory actions · 8 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed acquired hemophagocytic lymphohistiocytosis associated with malignant disease 4 milestones | |||||
| Refused (EMA) | EU (EMA) | — | acquired hemophagocytic lymphohistiocytosis associated with malignant disease | 2020-11-13 | “CHMP has adopted a negative opinion recommending a refusal of the marketing authorisation for emapalumab” sobi.com ↗ |
| Refused (EMA) | EU (EMA) | — | acquired hemophagocytic lymphohistiocytosis associated with malignant disease | 2020-07-24 | “CHMP has adopted a negative opinion recommending a refusal of the marketing authorisation for emapalumab” sobi.com ↗ |
| Filed | EU (EMA) | — | acquired hemophagocytic lymphohistiocytosis associated with malignant disease | — | “Novimmune submitted a Marketing Authorisation Application (MAA) for emapalumab to EMA in August 2018.” sobi.com ↗ |
| Filed | US (FDA) | — | acquired hemophagocytic lymphohistiocytosis associated with malignant disease | — | “Emapalumab application for US regulatory approval was filed with the FDA in March 2018” sobi.com ↗ |
| Filed hemophagocytic syndrome 1 milestone | |||||
| Filed | China (NMPA) | — | hemophagocytic syndrome | 2020-12-10 | “a Marketing Authorization Application for emapalumab has been accepted for review in China.” sobi.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| 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
News releases announcing trial results or a regulatory action · 20
| Date | Issuer | Release |
|---|---|---|
| 2026-03-18 | Sobi, Inc. | Results Results from EMBRACE Phase 2a study of emapalumab in interferon-gamma-driven sepsis presented at ISICEM sobi.com ↗ |
| 2026-02-18 | Sobi, Inc. | Results Sobi Capital Markets Day 2026: Bringing brilliant ideas to life and building the next chapter with ambition to deliver SEK 55bn by 2030 sobi.com ↗
High dose emapalumab additionally showed a 12-percentage point absolute mortality reduction after 28-days (40% mortality in the high dose emapalumab arm vs 52% in the standard of care plus placebo arm). |
| 2026-01-07 | Sobi, Inc. | Results Sobi to advance Gamifant® (emapalumab) in interferon-gamma-driven sepsis (IDS) based on EMBRACE topline data sobi.com ↗
Sobi® (STO: SOBI) today announced that topline results from the Phase 2a EMBRACE study evaluating Gamifant® (emapalumab) for interferon-gamma (IFNγ)-driven sepsis (IDS), showed proof-of-concept with observed improvement in organ dysfunction and survival. |
| 2025-06-27 | Sobi, Inc. | Regulatory FDA approves Gamifant® (emapalumab-lzsg) as first-ever treatment for adults and children with Macrophage Activation Syndrome in Still's disease sobi.com ↗ |
| 2025-02-27 | Sobi, Inc. | Regulatory US FDA grants Priority Review to Sobi's supplemental Biologics Licence Application (sBLA) for Gamifant ® (emapalumab-lzsg) sobi.com ↗
The application was granted Priority Review with a PDUFA date of June 27, 2025. |
| 2024-10-30 | Sobi, Inc. | Results New data for emapalumab in the treatment of macrophage activation syndrome to be presented at the ACR conference sobi.com ↗
At week 8, 21 patients (53.8%) achieved a complete response (95% confidence interval (CI): 37.2–69.9%). |
| 2022-03-18 | Sobi, Inc. | Regulatory Gamifant approved in China for the treatment of primary haemophagocytic lymphohistiocytosis sobi.com ↗
Sobi® today announced that the National Medical Products Administration of China (NMPA) has approved Gamifant® (emapalumab) for use in China. |
| 2022-02-25 | Sobi, Inc. | Regulatory Gamifant (emapalumab) recommended for approval in China for treatment of primary haemophagocytic lymphohistiocytosis (HLH) sobi.com ↗
Sobi® today announces that the Center for Drug Evaluation (CDE) has recommended approval of Gamifant® to the National Medical Products Administration of China (NMPA). |
| 2021-11-04 | Sobi, Inc. | Results Sobi to present new data at the ASH 2021 Annual Meeting sobi.com ↗
These data demonstrate the pathogenic role of IFNγ in MAS/sJIA and the therapeutic value of IFNγ neutralisation in patients with MAS who have failed standard therapy with high-dose glucocorticoids. |
| 2020-12-10 | Sobi, Inc. | Regulatory Emapalumab submission in China for primary HLH sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced that a Marketing Authorization Application for emapalumab has been accepted for review in China. |
| 2020-11-13 | Sobi, Inc. | Regulatory The CHMP has adopted a negative opinion for emapalumab in Europe for the treatment of primary HLH sobi.com ↗
The negative opinion was given after the re-examination requested by Sobi after the initial opinon in July 2020. |
| 2020-11-06 | Sobi, Inc. | Results Strong Sobi presence at American Society of Hematology 2020 Annual Meeting sobi.com ↗
Key results from the pivotal phase 2/3 study will be presented, with analyses confirming the study's primary endpoint and supporting a flexible dosing scheme with a favourable safety profile for emapalumab in patients with primary haemophagocytic lymphohistiocytosis. |
| 2020-10-17 | Sobi, Inc. | Results Emapalumab's efficacy confirmed by sensitivity analysis presented at ESID2020 sobi.com ↗ |
| 2020-07-24 | Sobi, Inc. | Regulatory Sobi will file for a re-examination of emapalumab in Europe following negative opinion by CHMP sobi.com ↗
Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announced that the Committee for Medicinal Products for Human use (CHMP) has adopted a negative opinion recommending a refusal of the marketing authorisation for emapalumab for the treatment of primary haemophagocytic lymphohistiocytosis (HLH) in children under 18 years of age in Europe. |
| 2020-05-07 | Sobi, Inc. | Results Results from pivotal phase 2/3 study of emapalumab in patients with primary HLH published in New England Journal of Medicine sobi.com ↗
Sobi™ announced today that the results from the pivotal phase 2/3 study evaluating the efficacy and safety of emapalumab in patients with primary haemophagocytic lymphohistiocytosis (HLH) were published in the New England Journal of Medicine on 7 May 2020. |
| 2019-07-17 | Sobi, Inc. | Results Sobi publishes report for second quarter 2019 sobi.com ↗
New data on emapalumab in patients with macrophage activation syndrome (MAS), a form of secondary HLH complicating systemic juvenile idiopathic arthritis (sJIA), showed that treatment with emapalumab led to a complete response in all si |
| 2019-06-18 | Sobi, Inc. | Results New data demonstrates impact of emapalumab in patients with macrophage activation syndrome (MAS) sobi.com ↗
The study showed that treatment with emapalumab led to rapid neutralisation of interferon gamma (IFNγ) and a complete response in all patients who participated in the study. |
| 2019-02-26 | Sobi, Inc. | Results New emapalumab data presented at Transplantation and Cellular Therapy (TCT) Meeting highlight post-transplant treatment outcomes in primary HLH sobi.com ↗
In this study, the majority of these very sick patients who were treated with emapalumab were able to reach transplant, engraft and survive, |
| 2018-12-04 | Sobi, Inc. | Results Data presented at ASH support emapalumab as an innovative, targeted therapeutic option for primary HLH sobi.com ↗
Across both cohorts, the overall response rate (ORR) was significantly higher than the pre-specified null hypothesis of 40 per cent. |
| 2018-11-20 | Sobi, Inc. | Regulatory FDA approves Gamifant® (emapalumab), the first and only treatment for primary haemophagocytic lymphohistiocytosis (HLH) sobi.com ↗ |
All press releases naming this drug 27 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 25 sources stand behind the page.
| Field | Value | Cited 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 ↗ |
| 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 ↗ |
| 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 Antibody” NCT01459562 ↗ |
| 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 |