Last reviewed 2026-04-19 · How we verify

Emapalumab-Lzsg

Emapalumab-Lzsg is a Interferon gamma Blocker [EPC] Small molecule drug developed by Swedish Orphan Biovitrum. It is currently in discontinued development for Primary hemophagocytic lymphohistiocytosis. Also known as: NI-0501.

12.1 Mechanism of Action Emapalumab-lzsg is a monoclonal antibody that binds to and neutralizes interferon gamma (IFNγ). Nonclinical data suggest that IFNγ plays a pivotal role in the pathogenesis of HLH by being hypersecreted.

At a glance

Mechanism of action

1 INDICATIONS AND USAGE GAMIFANT is indicated for the treatment of: adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. adult and pediatric ( newborn and older) patients with HLH/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. GAMIFANT is an interferon gamma (IFNγ) neutralizing antibody indicated for the treatment of: adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy. ( 1 ) adult and pediatric (newborn and older) patients with HLH/macrophage activation syndrome (MAS) in known or suspected Still’s disease, including systemic Juvenile Idiopathic Arthritis (sJIA), with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS. ( 1 )

Approved indications

• Primary hemophagocytic lymphohistiocytosis

Common side effects

No common side effects on file.

Drug interactions

• 7 DRUG INTERACTIONS 7.1 Effect of GAMIFANT on Cytochrome P450 Substrates The formation of CYP450 enzymes may be suppressed by increased levels of cytokines (such as IFNγ) during chronic inflammation. By neutralizing IFNγ, use of GAMIFANT may normalize CYP450 activities which may reduce the efficacy of drugs that are CYP450 substrates due to increased metabolism. Upon initiation or discontinuation of concomitant GAMIFANT, monitor for reduced efficacy and adjust dosage of CYP450 substrate drugs as appropriate.

Key clinical trials

• An Open Label, Single Arm, Multi-Centre, Post-authorization Study to Describe the Safety and Efficacy of Emapalumab for the Treatment of Primary Hemophagocytic Lymphohistiocytosis in Treatment Experie (Phase 4)
• Pilot Study of Emapalumab With Post-Transplant Cyclophosphamide as Graft-Versus-Host Disease Prophylaxis for Reduced-Intensity Allogeneic Hematopoietic Cell Transplantation (Phase 1)
• A Phase 2/3, Open-label, Single Arm, Multicenter Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Emapalumab in Adult Patients With Hemophagocytic Lymphohistiocytosis (Phase 2)
• A Two-cohort, Open-label, Single Arm, Multicenter Study to Evaluate Efficacy, Safety and Tolerability, PK and PD, of Emapalumab in Children and Adults With MAS in Still's Disease or With MAS in System (Phase 3)

Primary sources

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Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Emapalumab-Lzsg CI brief — competitive landscape report
• Emapalumab-Lzsg updates RSS · CI watch RSS
• Swedish Orphan Biovitrum portfolio CI

Frequently asked questions about Emapalumab-Lzsg

Related

• Drug class: All Interferon gamma Blocker [EPC] drugs
• Target: All drugs targeting Interferon gamma
• Manufacturer: Swedish Orphan Biovitrum — full pipeline
• Therapeutic area: All drugs in Immunology
• Indication: Drugs for Primary hemophagocytic lymphohistiocytosis
• Also known as: NI-0501
• Compare: Emapalumab-Lzsg vs similar drugs
• Pricing: Emapalumab-Lzsg cost, discount & access