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ORPHA:900

Granulomatosis with polyangiitis

medium confidenceDisorder

Also known as: GPA · Wegener granulomatosis

Publications

23,115

97.4th percentile

Trials

75

Interventional, condition-specific

Researchers

1,135

Distinct authors in sample

Gene link

—

Readiness

4/6

Stages with a signal

Clinical definition (Orphanet)

A rare anti-neutrophil cytoplasmic antibodies (ANCA)-associated vasculitis characterized by necrotizing inflammation of small and medium vessels (capillaries, venules and arterioles), resulting in tissue ischemia.

How rare: 1-9 / 100 000 — about one to nine people per hundred thousand.

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (6)

Wegener's granulomatosis · Wegener's syndrome · granulomatosis - Wegener's · granulomatosis with polyangiitis · necrotizing respiratory granulomatosis · pauci-immune glomerulonephritis associated with granulomatosis with polyangiitis

Research stages

Trial readiness signals

Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”

4/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    23,115 matched papers (11,782 in last 10 years) Source

  3. Phenotype characterisedPresent

    182 HPO annotations (e.g. Recurrent respiratory infections; Skin rash; Arthritis) Source

  4. Animal modelNot found

    No Alliance genotype “model of” associations via Monarch for these Mondo IDs

  5. Orphan designationPresent

    4 FDA · 2 EMA designations (3 FDA orphan-indication approvals) — e.g. avacopan Source

  6. Interventional trialPresent

    75 matched on ClinicalTrials.gov (13 recruiting in sample)

Biology

Genes and phenotypes

Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.

Do we know what causes it?

Not yet — the cause hasn't been pinned down in GenCC.

No strong gene–disease assertion joined for this Orphanet entity.

Phenotypes (Monarch / HPO)

182

Associated phenotypes · MONDO:0012105

  • Recurrent respiratory infections
  • Skin rash
  • Arthritis
  • Peripheral neuropathy
  • Scleritis

Showing 5 of 182 — open Monarch for the full list.

Animal models (Monarch / Alliance)

None returned for this Mondo ID. Empty here is not proof that no model organism work exists under another name or gene.

Monarch fetch 2026-07-29

Therapies

Designations, candidates, and chemicals

FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.

Orphan designation (FDA · EMA)

6

Designations · 3 with FDA orphan-indication approval

  • FDA avacopanChurg-Strauss Syndrome Vasculitides Microscopic Polyangiitis Granulomatosis with polyangiitis Wegener Granulomatosis · 2014-06-02 · Not FDA Approved for Orphan Indication
  • FDA gusperimus trihydrochlorideWegener's Granulomatosis · 2011-06-29 · Not FDA Approved for Orphan Indication
  • FDA EtanerceptWegener's Granulomatosis · 1999-04-06 · Not FDA Approved for Orphan Indication
  • EMA gusperimus trihydrochloride (Spanidin; Spanidin)Treatment of Wegener's granulomatosis · 29/03/2001 · PositiveEMA designation
  • EMA Vynpenta (previously AvacopanChemoCentryx); Tavneos (Vynpenta (previously AvacopanChemoCentryx); Tavneos)Treatment of granulomatosis with polyangiitis · 19/11/2014 · PositiveEMA designation
  • FDA rituximab (Rituxan)Churg-Strauss Syndrome Microscopic Polyangiitis Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis Wegener's Granulomatosis · 2006-02-14

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

31

Drugs / clinical candidates · MONDO_0012105

CTD chemicals (MyDisease.info)

19 associated chemicals · 19 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • Adrenal Cortex Hormones · therapeutic
  • Antilymphocyte Serum · therapeutic
  • Azathioprine · therapeutic
  • Cyclophosphamide · therapeutic
  • Cyclosporine · therapeutic
  • Etoposide · therapeutic
  • Glucocorticoids · therapeutic
  • Leflunomide · therapeutic
  • Mesna · therapeutic
  • Methotrexate · therapeutic
  • Methylprednisolone · therapeutic
  • Mycophenolic Acid · therapeutic

Pathways: Phagosome; Osteoclast differentiation; Natural killer cell mediated cytotoxicity; Leishmaniasis; Staphylococcus aureus infection; Tuberculosis; Systemic lupus erythematosus; Hemostasis

MyDisease.info · MONDO:0012105

Literature

Is anyone studying this?

23,115

23,115 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.

23,115 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).

11,782 in the last 10 years · medium confidence · 97.4th percentile (publications denominator)

Phrase hits: 23,115 · MeSH hits: 0

Open Europe PMC search

Who's working on it?

1,135

Distinct author names in 200 sampled papers — named people below.

Who's working on it?

People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.

  1. 01
    Emmi G4 papers · 2026

    Department of Medical, Surgery and Health Sciences, University of Trieste, Trieste, Italy.

    Papers in Europe PMC
  2. 02
    Avula S3 papers · 2026

    Internal Medicine, University of Minnesota, Minneapolis, USA

    Papers in Europe PMC
  3. 03
    Chen H3 papers · 2026

    Department of Pathology, Hospital for Skin Diseases, Institute of Dermatology, Chinese Academy of Medical Sciences & Peking Union Medical College, Nanjing, China. ch76ch@163.com.

    Papers in Europe PMC
  4. 04
    Higuchi T3 papers · 2026

    Department of Rheumatology, Tokyo Women's Medical University School of Medicine, Tokyo, Japan.

    Papers in Europe PMC
  5. 05
    Mohammad AJ3 papers · 2026

    Section of Rheumatology, Department of Clinical Sciences, Lund University, Lund, Sweden.

    Papers in Europe PMC
  6. 06
    Pagnoux C3 papers · 2026

    Vasculitis Clinic, Division of Rheumatology, Mount Sinai Hospital, University of Toronto, Toronto, ON, Canada.

    Papers in Europe PMC
  7. 07
    Ueki S3 papers · 2026

    Department of General Internal Medicine and Clinical Laboratory Medicine, Akita University Graduate School of Medicine, Japan.

    Papers in Europe PMC
  8. 08
    Zhou Y3 papers · 2026

    Department of Rheumatology, University of Wisconsin Health, Madison.

    Papers in Europe PMC
  9. 09
    Albayrak F2 papers · 2026

    Department of Rheumatology, Gaziantep University, Gaziantep, Turkey.

    Papers in Europe PMC
  10. 10
    Antiochos B2 papers · 2026

    Division of Rheumatology, Johns Hopkins University School of Medicine, Baltimore, MD.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

75

interventional trials for this specific condition

75 interventional trials matched this specific condition name; 13 currently recruiting in our sample.

Data as of 11 September 2026

75 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 98.1th percentile).

medium confidence · 98.1th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

75 interventional trials matched after quoted-phrase search and title/condition post-filter.

Observational and natural-history studies

42 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

Other registries (secondary)

Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.

raw 40 · after dedupe 40 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 40 · dropped 0 · fetched 2026-07-29

Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri

No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).

Uncertain / not reviewed (40)

Where to find support

Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.

Orphanet entry for Granulomatosis with polyangiitis — check Associations / patient organisations on that page.

India — NPRD

Last verified 2026-07-26

This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.

Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.

How we counted this

Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.

"Granulomatosis with polyangiitis" OR "Wegener granulomatosis" OR "Wegener's granulomatosis" OR "Wegener's syndrome" OR "granulomatosis - Wegener's" OR "necrotizing respiratory granulomatosis" OR "pauci-immune glomerulonephritis associated with granulomatosis with polyangiitis"

Run this search on Europe PMC

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Granulomatosis with polyangiitis" OR "Wegener granulomatosis" OR "Wegener's granulomatosis" OR "Wegener's syndrome" OR "granulomatosis - Wegener's" OR "necrotizing respiratory granulomatosis" OR "pauci-immune glomerulonephritis associated with granulomatosis with polyangiitis"

Interventional trials matched via: phrase (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 75 interventional · 42 observational · 0 expanded access. Only interventional studies enter the trial headline.

Query health: ok — strategies attempted: phrase; with hits: phrase

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: GPA

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • 1 synonym(s) dropped by stoplist (may under-count)
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-26T15:50:10.538Z