EMPAVELI (pegcetacoplan)
Empaveli treats adults living with paroxysmal nocturnal hemoglobinuria (PNH). It also helps patients 12 years and older who have C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN) by reducing proteinuria. This medication is used to manage the immune system overactivation that causes red blood cell destruction and kidney damage in these specific populations.
How EMPAVELI Works
This drug works by binding to the complement protein C3 and its activation fragment C3b, which regulates the cleavage of C3 and the production of downstream activation effectors. By acting early in the complement cascade, it controls both extravascular and intravascular hemolysis while decreasing the deposition of C3 fragments in the kidneys.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2021-05-14
- Patent Cliff
- 2038
- Routes
- SUBCUTANEOUS
- Dosage Forms
- SOLUTION
EMPAVELI Approval History
What EMPAVELI Treats
3 indicationsEMPAVELI is approved for 3 conditions since its original approval in 2021. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Paroxysmal Nocturnal Hemoglobinuria
- C3 Glomerulopathy
- Membranoproliferative Glomerulonephritis
EMPAVELI Boxed Warning
SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B [see Warnings and Precautions (5.1) ] . Life-threatening and fatal infections with encapsulated bacteria have occurred in patients treated with complement inhibitors. These infections may become rapidly life-threatening or fatal ...
WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B [see Warnings and Precautions (5.1) ] . Life-threatening and fatal infections with encapsulated bacteria 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 encapsulated bacteria at least 2 weeks prior to the first dose of EMPAVELI, unless the risks of delaying therapy with EMPAVELI outweigh the risk of developing a serious infection. Comply with the most current Advisory Committee on Immunization Practices (ACIP) recommendations for vaccinations against encapsulated 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 encapsulated bacteria. Patients receiving EMPAVELI are at increased risk for invasive disease caused by encapsulated bacteria, even if they develop antibodies following vaccination. Monitor patients for early signs and symptoms of serious infections and evaluate immediately if infection is suspected. Because of the risk of serious infections caused by encapsulated bacteria, EMPAVELI is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the EMPAVELI REMS [see Warnings and Precautions (5.2) ] . WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA See full prescribing information for complete boxed warning. EMPAVELI increases the risk of serious and life-threatening infections caused by encapsulated bacteria including Streptococcus pneumoniae , Neisseria meningitidis and Haemophilus influenzae type B. Complete or update vaccination for encapsulated bacteria
EMPAVELI Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to EMPAVELI
3 of 5FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
18 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT07214298 | I-4316824 NCI-2025-06680, I-4316824 | Ph 1, Ph 2 | recruiting | Pegcetacoplan in Combination With Modified FOLFIRINOX for the Treatment of Metastatic Pancreatic Ductal Adenocarcinoma |
| NCT04572854 NOBLE results posted | APL2-C3G-204 | Ph 2 | completed | Study Assessing the Safety and Efficacy of Pegcetacoplan in Post-Transplant Recurrence of C3G or IC-MPGN |
| NCT03531255 | APL2-307 | Ph 3 | active not recruiting | Pegcetacoplan Long Term Safety and Efficacy Extension Study |
| NCT05809531 VALE | APL2-C3G-314 2023-504625-39-00, U1111-1290-7826 | Ph 3 | active not recruiting | An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis |
| NCT04919629 | I 798120 NCI-2021-04265, I 798120 | Ph 2 | recruiting | APL-2 and Pembrolizumab Versus APL-2, Pembrolizumab and Bevacizumab Versus Bevacizumab Alone for the Treatment of Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer and Malignant Effusion |
| NCT07020832 | APL2-DGF-318 | Ph 3 | recruiting | A Phase 3 Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults at High Risk of Delayed Graft Function (DGF) Following Kidney Allograft Transplantation |
| NCT06722157 VERDANT | 1497-0002 U1111-1308-7923 | Ph 2 | active not recruiting | A Study to Test Whether BI 771716 Helps People With an Advanced Form of Age-related Macular Degeneration (AMD) Called Geographic Atrophy |
| NCT05067127 VALIANT results posted | APL2-C3G-310 | Ph 3 | completed | Phase III Study Assessing the Efficacy and Safety of Pegcetacoplan in Patients With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis |
| NCT05148299 results posted | Sobi.PEGCET-201 2023-510443-37-00, 2021-003157-27 | Ph 2 | completed | A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation |
| NCT05096403 results posted | Sobi.PEGCET-101 | Ph 3 | completed | A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD) |
| NCT04901936 | APL2-PNH-209 | Ph 2 | recruiting | A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| NCT03500549 results posted | APL2-302 | Ph 3 | completed | Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| NCT02588833 PADDOCK results posted | APL2-CP-PNH-204 | Ph 1 | completed | Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. |
| NCT02264639 results posted | APL-CP0514 | Ph 1 | completed | A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH |
| NCT03593200 results posted | APL2-202 | Ph 2 | completed | A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered Pegcetacoplan (APL-2) in Subjects With PNH |
| NCT02503332 FILLY results posted | POT-CP121614 | Ph 2 | completed | Study of Pegcetacoplan (APL-2) Therapy in Patients With Geographic Atrophy |
| NCT02461771 ASAP II results posted | POT-CP043014 | Ph 1 | completed | Assessment of Safety, Tolerability and Pharmacokinetics of Intravitreal Pegcetacoplan (APL-2) for Patients With Wet AMD |
| NCT03465709 results posted | APL2-203 | Ph 1, Ph 2 | terminated | Pegcetacoplan (APL-2) in Neovascular AMD |
Active Pipeline
Ongoing clinical trials by development phase
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.
EMPAVELI FDA Label Details
Indications & Usage
FDA Label (PDF)EMPAVELI is indicated for the treatment of Paroxysmal Nocturnal Hemoglobinuria; C3 Glomerulopathy; Membranoproliferative Glomerulonephritis.
WARNING: SERIOUS INFECTIONS CAUSED BY ENCAPSULATED BACTERIA EMPAVELI, a complement inhibitor, increases the risk of serious infections, especially those caused by encapsulated bacteria, such as Streptococcus pneumoniae , Neisseria meningitidis , and Haemophilus influenzae type B [see Warnings and Pr...
EMPAVELI Patents & Exclusivity
Patents (11 active)
Exclusivity
Pro Intelligence Preview
Deep insights for EMPAVELI
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2038
- • 27 active patents
Trial Analysis
- • 18 total trials
- • Stage: Expansion
Competitive Landscape
- • 5 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment