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About C3

Complement component 3 (C3) is a central protein in the complement system, enhancing antibody and phagocytic cell function to clear microbes and promote inflammation. As a key component of innate immunity, C3's dysregulation is implicated in various disorders.

2
Approved Drugs
1
Companies
5
Indications
2
Therapeutic Areas
Broadest Approval
EMPAVELI
APELLIS PHARMS
3
approved indications

Top C3 Drugs

EMPAVELI
APELLIS PHARMS
3 indications · 2021
SYFOVRE
APELLIS PHARMS
2 indications · 2023
🏢

Apellis Pharmaceuticals is the only company with approved C3-targeting drugs.

C3 Drug Modality Landscape

Modalities

Small molecule
2
100%

Routes of Administration

💉 Injection
1
50%
💧 Other
1
50%
💡

C3 is druggable by small molecules, though no oral formulations are currently approved.

Consider exploring alternative modalities like antibodies or biologics to differentiate from existing therapies.

Small molecules only

C3 Clinical Trials 28 trials

Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on C3. Source: ClinicalTrials.gov. How we count

28
Total Trials
10
Active
14
Completed
78%
Completion Rate

Completion by Phase

Phase Total Completed Stopped Active Completion
Phase 1 9 4 2 3 67%
Phase 2 9 4 1 4 80%
Phase 3 9 6 1 2 86%
Phase 4 1 0 0 1 -

Top Sponsors

Apellis Pharmaceuticals, Inc. 18 80%
Swedish Orphan Biovitrum 4 100%
Roswell Park Cancer Institute 2
Hoffmann-La Roche 1 0%
Columbia University 1
AbbVie 1
Boehringer Ingelheim 1

By Modality

Small molecule
28 78%
Source: ClinicalTrials.gov · Completion rate = completed ÷ (completed + terminated + withdrawn)

Phase 3 Readout Calendar Pro

2 Phase 3 trials testing approved C3 drugs across all sponsors.

Full calendar →
Q3 2027
Pegcetacoplan
Apellis Pharmaceuticals, Inc. · C3G
Estimated · aging NCT05809531
Q4 2025
Pegcetacoplan
Apellis Pharmaceuticals, Inc. · PNH
Completed · awaiting NCT03531255

Coverage: trials whose intervention is an approved drug targeting C3. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.

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Deep insights for drug target analysis

Competitive Landscape

  • • 1 companies competing
  • • Market share by company

Full Drug Portfolio

  • • All 2 approved drugs
  • • Approval dates & indications

Genetic Validation

  • • Full genetic evidence table
  • • Effect sizes & directions

Approval Timeline

  • • Full 2-drug timeline
  • • First-of-modality markers

Clinical Trials Analysis

  • • Sponsors: 7 (5 companies)
  • • Completion rates by condition
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Full summary • All drugs • Genetic evidence • Trials • Timeline

How We Count Trials

  • Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on C3. A trial counts if an approved drug acting on C3 is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
  • Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
  • Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
  • Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
  • Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.