EPYSQLI
EPYSQLI (eculizumab-aagh) is a complement inhibitor indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). It is also approved for use in adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive. The medication serves to reduce hemolysis in PNH and inhibit complement-mediated thrombotic microangiopathy in aHUS, though it is not indicated for Shiga toxin E. coli related hemolytic uremic syndrome.
How EPYSQLI Works
EPYSQLI is a monoclonal antibody that binds with high affinity to the complement protein C5, inhibiting its cleavage into C5a and C5b. This action prevents the generation of the terminal complement complex C5b-9, which otherwise mediates intravascular hemolysis in PNH and thrombotic microangiopathy in aHUS. In patients with gMG, the drug is presumed to work by reducing the deposition of this terminal complement complex at the neuromuscular junction.
Details
- Status
- Prescription
- First Approved
- 2024-07-19
- Routes
- SOLUTION
- Dosage Forms
- INJECTION
EPYSQLI Approval History
What EPYSQLI Treats
3 indicationsEPYSQLI is approved for 3 conditions since its original approval in 2024. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Paroxysmal Nocturnal Hemoglobinuria
- Atypical Hemolytic Uremic Syndrome
- Generalized Myasthenia Gravis
EPYSQLI Boxed Warning
SERIOUS MENINGOCOCCAL INFECTIONS Eculizumab products, complement inhibitors, increase the risk of serious infections caused by Neisseria meningitidis [ see Warnings and Precautions ( 5.1 ) ]. Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at...
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS Eculizumab products, complement inhibitors, increase the risk of serious infections caused by Neisseria meningitidis [ see Warnings and Precautions ( 5.1 ) ]. Life-threatening and fatal meningococcal infections have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal if not recognized and treated early. Complete or update vaccination for meningococcal bacteria (for serogroups A, C, W, Y, and B) at least 2 weeks prior to the first dose of EPYSQLI, unless the risks of delaying therapy with EPYSQLI outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against meningococcal bacteria in patients receiving a complement inhibitor. See Warnings and Precautions ( 5.1 ) for additional guidance on the management of the risk of serious infections caused by meningococcal bacteria. Patients receiving eculizumab products are at increased risk for invasive disease caused by Neisseria meningitidis , even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious meningococcal infections and evaluate immediately if infection is suspected. Because of the risk of serious meningococcal infections, EPYSQLI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called EPYSQLI REMS [ see Warnings and Precautions ( 5.2 ) ]. WARNING: SERIOUS MENINGOCOCCAL INFECTIONS See full prescribing information for complete boxed warning. Eculizumab products increase the risk of serious and life-threatening infections caused by Neisseria meningitidis. Complete or update meningococcal vaccination at least 2 weeks prior to the first dose of EPYSQLI, unless the risks of delaying EPYSQLI outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immuniz
EPYSQLI is a lower-cost alternative to Soliris with no clinically meaningful differences. Requires prescriber approval to substitute.
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Active Pipeline
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Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
EPYSQLI FDA Label Details
ProIndications & Usage
FDA Label (PDF)EPYSQLI is a complement inhibitor indicated for: the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy. Limitation of Use EPYSQLI is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). the treatment of generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AchR) antibody positive. 1.1 Paroxysmal Nocturnal Hemoglobinuria (PNH) EP...
WARNING: SERIOUS MENINGOCOCCAL INFECTIONS Eculizumab products, complement inhibitors, increase the risk of serious infections caused by Neisseria meningitidis [ see Warnings and Precautions ( 5.1 ) ]. Life-threatening and fatal meningococcal infections have occurred in patients treated with compleme...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.