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Pemphigus

GtoPdb Disease Summaries

This section gives an overview of the disease, and where available shows the following:

  • Synonyms: Shows known synonyms for the disease.
  • Description: Gives a basic description/definition of the disease.
  • Database Link: External links to the same disease at the Disease Ontology, OMIM or Orphanet sites.
  • Immunopharmacology comments: General comments about the target's role in immunopharmacology, provided by GtoImmuPdb curators.
  • Associated with: Counts are displayed for the total targets the disease is associated with in GtoPdb. The counts of targets and ligands of immunological relevance associated to the disease are also shown.

More information can be found in the help pages.

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Disease ID:1237
Name:Pemphigus
Associated with:1 target
1 immuno-relevant target
2 immuno-relevant ligands
Description
A group of rare, chronic autoimmune diseases of the skin and connective tissue which are characterized by severe blistering of the skin and mucous membranes. The most common subtype is pemphigus vulgaris (PV).
Database Links
Disease Ontology: DOID:9182

Targets

GtoPdb Disease Summaries - Targets

Click on the target name to link to its detailed view page

Where available, information is display on the role of the target in the disease; drugs which target the disease and their therapeutic use and side-effects.

If there is mutation data curated in GtoPdb this is indicated, with a link back to the appropriate section on the target detailed view page

Immuno ligand interactions - If available, a table of immuno-relevant ligands is shown. These ligands have been curated as having an association to the disease and possess interaction data with the target in GtoPdb. The approval status of the ligand is shown, along with curator comments and an indication of whether the target is considered the primary target of the ligand.

More information can be found in the help pages.

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Key to terms and symbols
CD20 (membrane-spanning 4-domains, subfamily A, member 1)
Comments:  CD20 is the molecular target of the antibody veltuzumab.
Ligand interactions: 
Ligand Comments
veltuzumab
FDA orphan drug for pemphigus.
rituximab
Rituximab is the first biologic therapy approved for pemphigus vulgaris (PV), and represents the first major PV therapeutic advance in more than 60 ye ...

Ligands

GtoPdb Disease Summaries - Ligands

Click ligand name to view ligand summary page

  • Approved: If the ligand is an approved drug this is indicated, along with approval bodies.
  • Immuno: Immuno icon indicates the ligand is immuno-relevant

Click the arrow in the final column to expand comments

  • Immuno Disease Comments: Curatorial comments specifically added as part of GtoImmuPdb. They give more information on the association between the ligand and disease in the context of immunopharmacology.
  • Clinical Use: General clinical comments relating to the ligand and may not necessarily be specific to the disease in question. With hyperlink to more details on the ligand summary pages.
  • Bioactivty Comments: Curatorial comments specifically about the compounds biological activity - with hyperlink to more details on the ligand summary pages.

More information can be found in the help pages.

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Key to terms and symbols Click ligand name to view ligand summary Click column headers to sort
Ligand References Clinical and Disease comments
veltuzumab
Immuno Disease Comments: FDA orphan drug for pemphigus.
Clinical Use: The EMA and FDA have granted veltuzumab orphan designation for the treatment of the rare disease chronic lymphocytic leukemia (CLL). The FDA also includes treatment of pemphigus (a group of autoimmune diseases causing severe blistering of the skin and mucous membranes) in its orphan drug designation. Phase 2 clinical trials for non-Hodgkin's lymphoma, CLL and autoimmune thrombocytopenic purpura are ongoing. | View clinical data
rituximab
Immuno Disease Comments: Rituximab is the first biologic therapy approved for pemphigus vulgaris (PV), and represents the first major PV therapeutic advance in more than 60 years.
Clinical Use: Used to treat CD20-positive non-Hodgkins lymphoma, chronic lymphocytic leukemia, and several autoimmune conditions (severe active, DMARD/TNF inhibitor-refracrtory rheumatoid arthritis; severe, active granulomatosis with polyangiitis (Wegener's, GPA); microscopic polyangiitis (MPA)). Rituximab is also used to suppress antibody-mediated rejection in living-donor kidney recipients prior to an ABO-incompatible transplant [3-4].
A modified formulation containing rituximab + human hyaluronidase (Rituxan Hycela) that can be delivered subcutaneously (the original rituximab only formulation has to be administered intravenously) was FDA approved in June 2017 for the treatment of previously untreated and relapsed or refractory follicular lymphoma, previously untreated diffuse large B-cell lymphoma (DLBCL), and previously untreated and previously treated chronic lymphocytic leukemia (CLL).
In June 2018, the FDA approved the use of rituximab as a much needed treatment for the potentially life-threatening skin disease pemphigus vulgaris (PV), for use in adult patients with moderate to severe disease. EMA approval for the treatment of PV patients followed in March 2019. | View clinical data
Bioactivity Comments: The patents covering rituximab do not contain any data regarding antibody-antigen affinity [1-2], but a dissociation constant is provided by Stein et al (2004) [5]. | View biological activity

References

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1. Anderson DR, Nabil H, Leonard JE, Newman RA, Reff ME, Rastetter WH. (1998) Therapeutic application of chimeric and radiolabeled antibodies to human B lymphocyte restricted differentiation antigen for treatment of B cell lymphoma. Patent number: US5843439. Assignee: Anderson DR, Nabil H, Leonard JE, Newman RA, Reff ME, Rastetter WH. Priority date: 13/11/1992. Publication date: 01/12/1998.

2. Anderson DR, Rastetter WH, Hanna N, Leonard JE, Newman RA, Reff ME. (1994) Therapeutic application of chimeric and radiolabeled antibodies to human b lymphocyte restricted differentiation antigen for treatment of b cell lymphoma. Patent number: WO1994011026. Assignee: Idec Pharma Corp. Priority date: 26/02/2015. Publication date: 26/05/1994.

3. Fehr T, Stussi G. (2012) ABO-incompatible kidney transplantation. Curr Opin Organ Transplant, 17 (4): 376-85. [PMID:22790072]

4. Macklin PS, Morris PJ, Knight SR. (2015) A systematic review of the use of rituximab as induction therapy in renal transplantation. Transplant Rev (Orlando), 29 (2): 103-8. [PMID:25555541]

5. Stein R, Qu Z, Chen S, Rosario A, Shi V, Hayes M, Horak ID, Hansen HJ, Goldenberg DM. (2004) Characterization of a new humanized anti-CD20 monoclonal antibody, IMMU-106, and Its use in combination with the humanized anti-CD22 antibody, epratuzumab, for the therapy of non-Hodgkin's lymphoma. Clin Cancer Res, 10 (8): 2868-78. [PMID:15102696]