Overview
Emapalumab, also known as NI-0501, is a fully human monoclonal antibody that targets interferon gamma. Emapalumab development was sponsored by NovImmune SA, further developed by Sobi and FDA approved on November 20, 2018. The approval of emapalumab was followed by the designation of orphan drug, priority review and breakthrough therapy. As well, emapalumab was given the status of PRIME by the EMA.
Indication
Emapalumab is indicated for the treatment of pediatric and adult patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance to conventional HLH therapy. The HLH condition is a hyperinflammatory status characterized by the overwhelming activation of normal T lymphocytes and macrophages which can lead to disturbances in the hematology profile and even death. As part of the condition profile, there have been reports proving a massive overexpression of interferon-gamma which is thought to drive the immune hyperactivation leading to organ failure. This condition is usually developed and present the symptomatic profile within the first months or years of life. These symptoms consist of fever, enlarged liver or spleen and a lower number of blood cells.
Associated Conditions
- Progressive, refractory, primary intolerance with conventional therapy, recurrent Hemophagocytic Lymphohistiocytosis
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2025/05/30 | Phase 1 | Not yet recruiting | City of Hope Medical Center | ||
2024/08/12 | Phase 2 | Recruiting | Marcela V. Maus, M.D.,Ph.D. | ||
2024/05/28 | Phase 2 | Recruiting | |||
2024/02/29 | Early Phase 1 | Recruiting | |||
2023/03/28 | Phase 2 | Recruiting | |||
2023/02/24 | Phase 4 | Active, not recruiting | |||
2021/08/12 | Phase 3 | Completed | |||
2021/02/21 | Phase 1 | Completed | |||
2021/01/29 | Phase 2 | Terminated | |||
2019/06/13 | Phase 2 | Terminated |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| Swedish Orphan Biovitrum AB (publ) | 66658-505 | INTRAVENOUS | 50 mg in 10 mL | 6/21/2023 | |
| Swedish Orphan Biovitrum AB (publ) | 66658-524 | INTRAVENOUS | 250 mg in 10 mL | 6/21/2023 | |
| Swedish Orphan Biovitrum AB (publ) | 66658-510 | INTRAVENOUS | 100 mg in 20 mL | 6/21/2023 | |
| Swedish Orphan Biovitrum AB (publ) | 66658-523 | INTRAVENOUS | 100 mg in 4 mL | 6/21/2023 | |
| Swedish Orphan Biovitrum AB (publ) | 66658-501 | INTRAVENOUS | 10 mg in 2 mL | 6/21/2023 | |
| Swedish Orphan Biovitrum AB (publ) | 66658-522 | INTRAVENOUS | 50 mg in 2 mL | 6/21/2023 | |
| Swedish Orphan Biovitrum AB (publ) | 66658-525 | INTRAVENOUS | 500 mg in 20 mL | 6/21/2023 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
| No EMA approvals found for this drug. | |||
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No HSA approvals found for this drug. | |||||
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| No TGA approvals found for this drug. | |||||
Health Canada Drug Approvals
Approved Product | Company | DIN | Dosage Form | Strength | Market Date |
|---|---|---|---|---|---|
| No Health Canada approvals found for this drug. | |||||
CIMA AEMPS Drug Approvals
Approved Product | Company | Registration Number | Pharmaceutical Form | Prescription Type | Status |
|---|---|---|---|---|---|
| No CIMA AEMPS (Spain) approvals found for this drug. | |||||
Philippines FDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Philippines FDA approvals found for this drug. | |||||
Saudi SFDA Drug Approvals
Approved Product | Company | License Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Saudi SFDA approvals found for this drug. | |||||
Malaysia NPRA Drug Approvals
Approved Product | Company | Registration Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No Malaysia NPRA approvals found for this drug. | |||||
UK EMC Drug Information
Medicine Name | MA Holder | MA Number | Pharmaceutical Form | Active Ingredient | Authorization Date |
|---|---|---|---|---|---|
| No UK EMC drug information found for this drug. | |||||
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