Drug discovery
21
drugs
With orphan designations
Overview
Juvenile idiopathic arthritis (JIA) is a chronic autoimmune disease characterized by persistent joint inflammation (≥6 weeks) in children <16 years, with subtypes classified by clinical features. Diagnosis requires exclusion of alternative causes and may involve elevated inflammatory markers (ESR/CRP) or imaging. Early aggressive therapy with DMARDs/biologics improves remission rates, while untreated cases risk joint damage, growth impairment, and uveitis (15-30% risk) [1][6][11][17]. Management requires multidisciplinary care to address physical, ocular, and psychosocial impacts [1][16].
Population
Burden
25% of adults with JIA require biologics; 44% use glucocorticoids long-term [10][14]
Systemic JIA caregivers lose 25 workdays/year; patients miss 12% of school yearly [4][9]
31% with JIA have concurrent anxiety/depression; 19-20% higher risk of sustained high disease activity with comorbid joint hypermobility [12][15]
Therapies
First-line: Methotrexate ± intra-articular steroids (65% remission in oligoarticular JIA) [8][18]
Biologic escalation: Anti-IL1 (anakinra) or anti-IL6 (tocilizumab) for systemic JIA; TNF inhibitors for polyarticular forms [3][6][13]
Treat-to-target protocols achieve clinical inactivity in 66% within 12 months when initiated early [3][18]
Categories: rare respiratory diseases, rare systemic and rheumatological diseases, rare systemic or rheumatologic diseases of childhood, rare transplant-related disorders
Drug Discovery Landscape
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
sirukumab | antibodies | FDA | 2017-07-03 | — | Janssen Research & Development, LLC |
protoplasmic protein matrix, omega-3 fatty oils, vitamins, minerals and trace elements, cell membrane matrix phospholipid, entracellular matrix, lipids, medium chain triglycerides, longer chain triglycerides, carbohydrates, probiotic, and phytonutrients | other | FDA | 2017-01-11 | — | Leonard S. Girsh, MD |
Celecoxib oral liquid suspension | small molecules | FDA | 2017-01-05 | — | NuBioPharma, LLC |
deflazacort | small molecules | FDA | 2015-10-22 | — | PTC Therapeutics, Inc. |
upadacitinib [Rinvoq] | small molecules | FDA | 2015-09-18 | 2024-04-26 | AbbVie, Inc. |
Kre-Celazine | small molecules | FDA | 2013-04-01 | — | All American Pharmaceutical & Natural Foods Corpor |
Givinostat | small molecules | EMA | 2010-01-28 | — | Italfarmaco S.p.A. |
canakinumab [ILARIS] | antibodies | FDA | 2008-09-30 | 2013-05-09 | Novartis Pharmaceuticals Corporation |
nabumetone | small molecules | FDA | 2008-05-05 | — | Cook Pharma |
adalimumab [Humira] | antibodies | FDA | 2005-03-21 | 2008-02-21 | AbbVie Inc. |
rofecoxib | small molecules | FDA | 2004-03-16 | — | MERCK & Co., Inc. |
meloxicam [Mobic] | small molecules | FDA | 2002-11-22 | 2005-08-11 | Avondale Pharmaceuticals |
infliximab | antibodies | FDA | 2002-10-23 | — | Centocor, Inc. |
human gammaglobulin | antibodies | FDA | 2001-05-25 | — | Latona Life Sciences, Inc. |
etanercept [Enbrel] | proteins | FDA | 1998-10-27 | 1999-05-27 | Immunex Corporation |
interferon Beta-1a | proteins | FDA | 1998-10-14 | — | Biogen, Inc. |
Purified type II collagen | proteins | FDA | 1995-02-09 | — | AutoImmune, Inc. |
Gammalinolenic acid | small molecules | FDA | 1994-07-27 | — | Zurier, Robert B. M.D. |
methotrexate | small molecules | FDA | 1993-08-23 | — | Wyeth-Ayerst Laboratories |
Immune globulin intravenous (human) | antibodies | FDA | 1992-12-16 | — | Immuno Clinical Research Corp. |
Interleukin-1 receptor antagonist, human recombinant | proteins | FDA | 1991-09-23 | — | Swedish Orphan Biovitrum AB (publ) (SOBI) |