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Protein / target

Complement factor D

Encoded byCFDP00746Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
1
Approved medicines
Open Targets target-level
View by indication →
23
Clinical trials
Small-molecule tractable
Druggability
Advanced Clinical

Protein at a glance

Biological role

Serine-type endopeptidase

Strongest disease association

Gastrointestinal disease

Via encoding gene CFD · Genetic evidence · score 0.49

Therapeutic position

Established drug target

Small molecules and antibodies

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function overview

Serine protease that initiates the alternative pathway of the complement system, a cascade of proteins that leads to phagocytosis and breakdown of pathogens and signaling that strengthens the adaptive immune system.

View complete UniProt function annotation

Serine protease that initiates the alternative pathway of the complement system, a cascade of proteins that leads to phagocytosis and breakdown of pathogens and signaling that strengthens the adaptive immune system (PubMed:21205667, PubMed:22362762, PubMed:6769474, PubMed:874324, PubMed:9748277). In contrast to other complement pathways (classical, lectin and GZMK) that are directly activated by pathogens or antigen-antibody complexes, the alternative complement pathway is initiated by the spontaneous hydrolysis of complement C3 (PubMed:21205667, PubMed:22362762, PubMed:6769474, PubMed:874324). The alternative complement pathway acts as an amplification loop that enhances complement activation by mediating the formation of C3 and C5 convertases (PubMed:21205667, PubMed:22362762, PubMed:6769474, PubMed:874324). Activated CFD cleaves factor B (CFB) when the latter is complexed with complement C3b, activating the C3 convertase of the alternative pathway (PubMed:21205667, PubMed:6769474, PubMed:874324, PubMed:9748277)

Subcellular location

Secreted
Domains and Gene Ontology detail (14)

Domains & features

Peptidase S1

Gene Ontology

  • Cextracellular exosome
  • Cextracellular region
  • Cextracellular space
  • Cficolin-1-rich granule lumen
  • Cplatelet alpha granule lumen
  • Csecretory granule lumen
  • Fserine-type endopeptidase activity
  • Fserine-type peptidase activity
  • Pcomplement activation
  • Pcomplement activation, alternative pathway
  • Pprotein maturation
  • Pproteolysis

253 aa · 27 kDa

Biological roles

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

ProteolysisUniProt · GO
View supporting evidence

Proteolysis

  • ·Serine protease that initiates the alternative pathway of the complement system, a casca…
  • ·serine-type endopeptidase activity
  • ·serine-type peptidase activity
  • ·proteolysis

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Approved medicines with mapped indications

1 medicine · 2 areas

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Hemoglobinuria, Paroxysmal1 medicine
Hemolysis1 medicine

Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

1

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

danicopan
Narrow target profileApprovedInhibitor

Complement factor D inhibitor

Indicated for Hemoglobinuria, Paroxysmal, Hemolysis

Direct interaction with this protein · Only this protein recorded as a target

ChEMBL mechanism, action type, target identity and approved indications. Open Targets clinical status.

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene CFD

Gene-level evidence surfaced through the gene CFDthat encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Macular Degeneration
0.66Moderately supported

Genetic evidence dominant · Open Targets 0.29

Gastrointestinal disease
0.48Limited support

Genetic evidence dominant · Open Targets 0.29

Neurodegenerative Diseases
0.21Preliminary

Pathway evidence dominant · Open Targets 0.32 · no direct causal or clinical evidence

View evidence synthesis (3)
Macular DegenerationModerately supported
0.66
agreement 0.550.77
Genetic53%Clinical40%Literature7%

Open Targets aggregate 0.29 · 3 independent evidence families

Gastrointestinal diseaseLimited support
0.48
agreement 0.360.60
Genetic100%

Open Targets aggregate 0.29 · 1 independent evidence family

Neurodegenerative DiseasesPreliminary
0.21
agreement 0.000.44
Pathway100%

Open Targets aggregate 0.32 · 1 independent evidence family · no direct causal or clinical evidence

This ranking differs from Open Targets' own: re-weighting moves genetically-evidenced diseases above more heavily co-mentioned ones. The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the per-type scores above show the calculation.

Show all associations
Neurodegenerative Diseases0.32
Gastrointestinal disease0.29
Macular Degeneration0.29

Drug development

2 compounds recorded · 1 approved · 1 in clinical development

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 1 drug that targets this protein in Forefront's canonical graph (1 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (2)
LAMPALIZUMABPhase 3
DANICOPANApproval

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small moleculesStrong

Advanced Clinical and Structure with Ligand support this modality.

AntibodiesStrong

Advanced Clinical and UniProt loc high conf support this modality.

Protein degradersEmerging

Feasibility evidence (database ubiquitination and small molecule binder) — no clinical-stage drug of this modality recorded.

View underlying tractability evidence (10)
SM · Advanced ClinicalSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketAB · Advanced ClinicalAB · UniProt loc high confAB · GO CC high confAB · UniProt SigP or TMHMMPR · Database UbiquitinationPR · Small Molecule Binder

Raw Open Targets tractability assessment buckets, by modality.

Clinical trials

23

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

View all trials (19)

ClinicalTrials.gov via the drug-target graph.

What's happening now

2

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for a drug that targets this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

  1. Regulatory approval2024-04-19

    Approval: Voydeya (EMA)

    ema · regulatory · ema · via danicopan

  2. New publication2023-12-01
    Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial.

    The Lancet. Haematology · 2023 · 68 citations · Europe PMC · via danicopan

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.