Pegcetacoplan is a Complement C3 inhibitor approved for conditions such as paroxysmal nocturnal hemoglobinuria (PNH) and geographic atrophy secondary to age-related macular degeneration. This page compares Pegcetacoplan to other agents in its class, including iptacopan (Fabhalta). Pegcetacoplan's current approvals encompass a wider spectrum of complement-mediated diseases, including C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), in addition to PNH and geographic atrophy.
Pegcetacoplan Alternatives: How It Compares to Other Complement Inhibitors
Hipa.ai Research · Source: ClinicalTrials.gov / AACT · Last updated: AI-augmented data · 0/2 curated
Source: ClinicalTrials.gov via AACT · Hipa.ai, 2026-05-07Download chart as PNG
Pegcetacoplan, approved in 2021, precedes Fabhalta (iptacopan), which received approval in 2023, with no other Phase 3 pipeline drugs currently identified.
Quick comparison table
| Drug | Class | Approved indications | Dosing | Year approved | Lead pivotal endpoint | Annual cost (rough) |
|---|---|---|---|---|---|---|
| Pegcetacoplan (Empaveli, Syfovre) | Complement C3 inhibitor | Paroxysmal nocturnal hemoglobinuria (PNH), Geographic atrophy secondary to age-related macular degeneration, C3 glomerulopathy (C3G), +1 more | 1,080 mg twice weekly via subcutaneous infusion (Empaveli); intravitreal injection (Syfovre) | 2021 | Urine protein-to-creatinine ratio (UPCR) reduction: 68.3% @ 26 weeks | $517k |
| Iptacopan (Fabhalta) | Complement factor B inhibitor | Paroxysmal nocturnal hemoglobinuria, IgA nephropathy, C3 glomerulopathy | 200 mg twice daily | 2025 | UPCR reduction: 35.1% @ 6 months | $550k |
Cost estimates are list-price approximations and do not reflect rebates, formulary tier, or out-of-pocket costs after benefits. The class-typical lead-pivotal endpoint here is Proteinuria reduction; cells render each drug's actual pivotal endpoint, which may differ. The "Year approved" column shows the FDA approval year for C3 glomerulopathy specifically — drugs approved for other indications first appear with their this-indication date, or as Pipeline if not yet approved for this indication. Cross-trial comparisons can mislead — head-to-head Phase-3 data (when present) is below.
Pegcetacoplan vs Iptacopan (Fabhalta)
No head-to-head Phase-3 trial directly compares Pegcetacoplan with Iptacopan.
In separate pivotal trials, Pegcetacoplan reported 68.3% Urine protein-to-creatinine ratio (UPCR) reduction at 26 weeks (NCT05067127) versus 35.1% UPCR reduction at 6 months for Iptacopan (NCT04817618).
Cross-trial caveat: the two drugs were tested in different patient populations at different time points. Cross-trial comparisons of response rates can mislead — the only rigorous comparison is a head-to-head randomized trial.
Choosing between Pegcetacoplan and its alternatives
Clinicians considering complement inhibitors may weigh the distinct mechanisms of action. Pegcetacoplan targets complement C3, a central component of the complement cascade. This broad inhibition may be a factor in selecting treatment, particularly when a comprehensive blockade of both proximal and distal pathways is sought. Its mechanism offers a unique approach to complement modulation, which may be preferred in certain clinical scenarios.
In contrast, iptacopan (Fabhalta) acts as a factor B inhibitor, offering a more targeted blockade upstream in the alternative pathway. Clinical data for iptacopan has shown a mean urine protein-to-creatinine ratio (UPCR) reduction of 35.1% at 6 months in studies. Its dosing regimen of 200 mg twice daily provides a specific administration schedule. The choice between these agents may also involve considerations such as a drug's longer track record in specific patient populations, cost implications, or a safety profile that aligns better with individual patient needs, particularly when a more targeted complement inhibition strategy is preferred due to its specific mechanism.
Ultimately, the selection of a complement inhibitor is a complex clinical decision that must be individualized for each patient, taking into account their specific condition, comorbidities, and treatment goals. This information is not medical advice, and clinical decisions should always be made by a qualified prescriber.
Sources and methodology
Trial data was pulled from the ClinicalTrials.gov registry via the AACT relational mirror maintained by the Clinical Trials Transformation Initiative. AACT data freshness: .
Cross-trial comparison limitations:drugs without a direct head-to-head trial are compared using each drug's own pivotal trial. These trials enrolled different patient populations at different time points and used different statistical analysis sets. Cross-trial response-rate differences should not be interpreted as proof that one drug is more effective than another.
Related drug pages on Hipa.ai
Not medical advice. This page summarizes publicly-reported clinical trial data for informational purposes. Treatment decisions belong with a qualified prescribing clinician who knows your medical history. Drug approvals, dosing, and safety profiles change over time — always confirm with the current FDA prescribing information.
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