

Drug discovery
6
drugs
With orphan designations
Overview
Polymyositis (PM) is a rare idiopathic inflammatory myopathy characterized by autoimmune-mediated skeletal muscle inflammation, leading to progressive symmetric proximal muscle weakness. Symptoms include difficulty climbing stairs, rising from seated positions, dysphagia, and fatigue. Systemic complications may involve interstitial lung disease, cardiomyopathy, or arthritis. Diagnosis requires clinical evaluation, elevated muscle enzymes (e.g., CK), electromyography, MRI, and muscle biopsy [1][5][13].
Population
Prevalence: 5–22 cases per 100,000 individuals globally, with peak incidence in adults aged 50–70 [2][4].
Gender: Female predominance (2:1 ratio), higher prevalence in African Americans [17][12].
Comorbidities: Associated with malignancies (e.g., lung, breast) and connective tissue diseases (e.g., lupus, scleroderma) [9][11].
Burden
Survival: 75% 5-year survival (PM) vs. 63% (dermatomyositis); median survival 11–12 years [2][4].
Complications: Respiratory failure, dysphagia-related aspiration, and malignancy-driven mortality [9][17].
Healthcare impact: High disability rates, prolonged immunosuppressive therapy, and frequent hospitalizations [9][19].
Therapies
First-line: High-dose corticosteroids (prednisone) ± steroid-sparing immunosuppressants (methotrexate, azathioprine) [3][8].
Refractory cases: Biologics (rituximab), IV immunoglobulin, or mycophenolate mofetil [3][18].
Supportive care: Physical therapy to preserve muscle function and multidisciplinary management for dysphagia/respiratory involvement [3][13].
Categories: rare neurological diseases, rare renal diseases, rare systemic and rheumatological diseases, rare transplant-related disorders
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Glucagon like peptide-1 (GLP-1) Elastin like peptide (ELP)-120 fusion protein | proteins | FDA | 2021-08-31 | — | ImmunoForge Co., Ltd. |
(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 | small molecules | FDA | 2020-10-21 | — | Kezar Life Sciences, Inc. |
Siponimod [BAF312] | small molecules | EMA | 2014-11-19 | — | Novartis Europharm Limited |
Siponimod | small molecules | FDA | 2013-11-26 | — | Novartis Pharmaceuticals Corporation |
Human normal immunoglobulin [Gammagen] | antibodies | EMA | 2003-10-24 | — | [INACTIVE] Orfagen |
Immune globulin intravenous (human) | antibodies | FDA | 1992-10-13 | — | Immuno Clinical Research Corp. |