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A rare idiopathic inflammatory myopathy characterized by symmetric proximal muscle weakness and elevated muscle enzymes.
Biomarker and diagnostic research for polymyositis has been reported in the published literature.
No approved treatments are currently available for polymyositis. An additional 3 compounds hold orphan drug designation.
While no drugs are FDA-approved specifically for polymyositis, 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 polymyositis. Orphan designation reflects regulatory interest and does not indicate approval for treatment.
Brand Name | Generic Name | Sponsor |
|---|
Estimated prevalence: 1-9 in 100,000 (Uncommon).
20 clinical trials registered, 12 recruiting. Interventions under study include other interventions, biologic therapy, drug therapy, and procedural interventions. Pipeline includes 4 PHASE3, 6 PHASE2, 4 PHASE1. Research is sponsored by a mix of industry and academic institutions.
NCT ID | Title | Phase | Sponsor | Status |
|---|---|---|---|---|
[NCT05895786](https://clinicaltrials.gov/study/NCT05895786) |
Data assembled from 5 of 12 sources · Last updated Sep 17, 2026, 9:26 PM UTC
European rare disease database
Genetic and Rare Diseases Info Center
Designated
Exclusivity End |
|---|
Designation Status |
|---|
Glucagon like peptide-1 (GLP-1) Elastin like peptide (ELP)-120 fusion protein | Glucagon like peptide-1 (GLP-1) Elastin like peptide (ELP)-120 fusion protein | ImmunoForge Co., Ltd. | 2021 | — | Designated |
(2S,3R)-N-[(2S)-3-(cyclopent-1-en-1-yl)-1-[(2R)-2-methyloxiran-2-yl]-1-oxopropan-2-yl]-3-hydroxy-3-(4-methoxyphenyl)-2-[(2S)-2-[2-(morpholin-4-yl)acetamido]propanamido]propanamide maleate | (2S,3R)-N-[(2S)-3-(cyclopent-1-en-1-yl)-1-[(2R)-2-methyloxiran-2-yl]-1-oxopropan-2-yl]-3-hydroxy-3-(4-methoxyphenyl)-2-[(2S)-2-[2-(morpholin-4-yl)acetamido]propanamido]propanamide maleate | Kezar Life Sciences, Inc. | 2020 | — | Designated |
Siponimod | Siponimod | Novartis Pharmaceuticals Corporation | 2013 | — | Withdrawn |
Gene therapy approaches for polymyositis have been reported in the published literature.
20 trials found
A Study to Understand How the Study Medicine (PF-06823859) Works in People With Active Idiopathic Inflammatory Myopathies [Dermatomyositis (DM) and Polymyositis (PM)] |
PHASE3 |
Pfizer |
RECRUITING |
[NCT06455449](https://clinicaltrials.gov/study/NCT06455449) | A Study to Investigate the Efficacy and Safety of Anifrolumab Administered as Subcutaneous Injection and Added to Standard of Care Compared With Placebo Added to Standard of Care in Adult Participants With Idiopathic Inflammatory Myopathies (Polymyositis and Dermatomyositis) | PHASE3 | AstraZeneca | RECRUITING |
[NCT07160205](https://clinicaltrials.gov/study/NCT07160205) | Safety and Efficacy of ULSC on Disease Severity and Steroid Tapering in Participants With Dermatomyositis/ Polymyositis (DM/PM), Also Known as Idiopathic Inflammatory Myopathy (IIM) | PHASE2 | Restem, LLC. | RECRUITING |
[NCT06154252](https://clinicaltrials.gov/study/NCT06154252) | RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy | PHASE2 | Cabaletta Bio | RECRUITING |
[NCT01276470](https://clinicaltrials.gov/study/NCT01276470) | Environmental Risk Factors for the Anti-synthetase Syndrome | — | National Institute of Environmental Health Sciences (NIEHS) | RECRUITING |
361 publications have been identified in PubMed for polymyositis. Kisho has analyzed 187 by research type. Research spans Review / Meta-Analysis (26%), Epidemiology / Natural History (26%), and Basic Science / Preclinical (18%).
Research Type | Count | % of Total |
|---|---|---|
Research summaries | 48 | 26% |
Disease patterns and progression | 48 | 26% |
Laboratory research | 33 | 18% |
Testing and diagnosis research | 23 | 12% |
Patient case studies | 23 | 12% |
Clinical study results | 7 | 4% |
Other research | 3 | 2% |
New treatment approaches | 2 | 1% |
Cheeti A (2026). [PMID: 30422455](https://pubmed.ncbi.nlm.nih.gov/30422455/). *Unknown Journal*. [Epidemiology / Natural History]
Guo J (2026). [PMID: 41481958](https://pubmed.ncbi.nlm.nih.gov/41481958/). *Int Immunopharmacol*. [Basic Science / Preclinical]
Han S (2026). [PMID: 41485053](https://pubmed.ncbi.nlm.nih.gov/41485053/). *Mol Ther*. [Basic Science / Preclinical]
Nosal RS (2026). [PMID: 30725756](https://pubmed.ncbi.nlm.nih.gov/30725756/). *Unknown Journal*. [Diagnostic / Biomarker]
Wilkerson JC (2026). [PMID: 41317376](https://pubmed.ncbi.nlm.nih.gov/41317376/). *Rheumatology (Oxford)*. [Review / Meta-Analysis]
Liu Y (2026). [PMID: 41466027](https://pubmed.ncbi.nlm.nih.gov/41466027/). *Brain Behav*. [Basic Science / Preclinical]
Kitahara Y (2026). [PMID: 41577954](https://pubmed.ncbi.nlm.nih.gov/41577954/). *Sci Rep*. [Diagnostic / Biomarker]
Ashe B (2026). [PMID: 42148098](https://pubmed.ncbi.nlm.nih.gov/42148098/). *Front Immunol*. [Case Report / Case Series]
Marwa K (2026). [PMID: 34283427](https://pubmed.ncbi.nlm.nih.gov/34283427/). *Unknown Journal*. [Epidemiology / Natural History]
Leclair V (2026). [PMID: 41206056](https://pubmed.ncbi.nlm.nih.gov/41206056/). *Rheumatology (Oxford)*. [Diagnostic / Biomarker]
AI-curated news mentioning polymyositis
Updated Jan 28, 2026
The MIHRA initiative focuses on gathering patient-rooted insights to shape research in myositis and related conditions. Sponsored by multiple organizations, including Myositis International and The Myositis Association, this project emphasizes qualitative investigations to better understand patient experiences.