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A hereditary palmoplantar keratoderma characterized by the combination of bilateral mutilating transgredient palmoplantar keratoderma and periorificial keratotic plaques.
No approved treatments are currently available for Olmsted syndrome. An additional 2 compounds hold orphan drug designation.
While no drugs are FDA-approved specifically for Olmsted syndrome, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.
The following drugs have received orphan drug designation from the FDA for Olmsted syndrome. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
Estimated prevalence: <1 in 1,000,000 (VERY_RARE).
1 clinical trial registered. Interventions under study include drug therapy. Pipeline includes 1 PHASE1. Research is primarily sponsored by academic and government institutions.
28 publications have been identified in PubMed for Olmsted syndrome. Research spans Case Report / Case Series (50%), Basic Science / Preclinical (29%), and Review / Meta-Analysis (7%).
Research Type | Count | % of Total |
|---|---|---|
Patient case studies | 14 | 50% |
Data assembled from 5 of 12 sources · Last updated Sep 19, 2026, 11:54 AM UTC
European rare disease database
Genetic and Rare Diseases Info Center
Common questions about Olmsted syndrome
Designated
Exclusivity End |
|---|
Designation Status |
|---|
(R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl)pyrrolidin-1-yl)(5-hydroxy-6-methylpyridin-2-yl)methanone | (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl)pyrrolidin-1-yl)(5-hydroxy-6-methylpyridin-2-yl)methanone | Kamari Pharma Ltd. | 2024 | — | Designated |
(R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone | (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone | Kamari Pharma Ltd. | 2023 | — | Designated |
Gene therapy approaches for Olmsted syndrome have been reported in the published literature.
1 trial found
Laboratory research |
8 |
29% |
Research summaries | 2 | 7% |
Other research | 1 | 4% |
Clinical study results | 1 | 4% |
Disease patterns and progression | 1 | 4% |
New treatment approaches | 1 | 4% |
Jiang X (2026). [PMID: 41533385](https://pubmed.ncbi.nlm.nih.gov/41533385/). *JAMA dermatology*. [Basic Science / Preclinical]
Sadeghzadeh-Bazargan A (2026). [PMID: 41362860](https://pubmed.ncbi.nlm.nih.gov/41362860/). *JAAD case reports*. [Case Report / Case Series]
Holloway A (2026). [PMID: 41787805](https://pubmed.ncbi.nlm.nih.gov/41787805/). *Biophysical journal*. [Basic Science / Preclinical]
Pereira da Conceição R (2026). [PMID: 40859917](https://pubmed.ncbi.nlm.nih.gov/40859917/). *International journal of dermatology*. [Clinical Trial Publication]
Azulay-Abulafia L (2026). [PMID: 41895212](https://pubmed.ncbi.nlm.nih.gov/41895212/). *An Bras Dermatol*. [Case Report / Case Series]
Chattapadhya A (2026). [PMID: 41932384](https://pubmed.ncbi.nlm.nih.gov/41932384/). *Life Sci*. [Basic Science / Preclinical]
Mo R (2025). [PMID: 39668001](https://pubmed.ncbi.nlm.nih.gov/39668001/). *Journal of dermatological science*. [Other]
Diversi F (2025). [PMID: 40171279](https://pubmed.ncbi.nlm.nih.gov/40171279/). *Molecular therapy. Nucleic acids*. [Gene Therapy / Novel Therapeutics]
Zhang X (2025). [PMID: 40615090](https://pubmed.ncbi.nlm.nih.gov/40615090/). *Journal of the American Academy of Dermatology*. [Case Report / Case Series]
Segal NL (2025). [PMID: 40922491](https://pubmed.ncbi.nlm.nih.gov/40922491/). *Twin research and human genetics : the official journal of the International Society for Twin Studies*. [Review / Meta-Analysis]