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Table 2 Orphan drugs for the treatment of rare rheumatologic disorders approved by the FDA between 1983 and 2013

From: Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act

Compound Approved indication according to FDA label Pharmacologic category Designation year Approval year Pivotal clinical trial Reference
Study design N Age Study duration Main outcome measure
Pilocarpine hydrochloride Treatment of dry mouth in patients with Sjögren’s syndrome Cholinergic agonist 1992 1998 RCT 256 mean 57 years (24 – 85) 12 weeks Global improvement of dry mouth [26]
RCT 373 mean 55 years (21 – 84) 12 weeks Global improvement of dry mouth
Etanercept Reduction in signs and symptoms of moderately to severely active polyarticular-course juvenile rheumatoid arthritis in patients who have had an inadequate response to one or more disease-modifying anti-rheumatic drugs Tumor necrosis factor-alpha inhibitor 1998 1999 RCT 69 2 – 17 years 7 months JIA definition of improvement criteria [10]
Dexamethasone (intravitreal implant) Treatment of non-infectious uveitis affecting the posterior segment of the eye Glucocorticoid 1998 2010 RCT 153 n/a 8 weeks Vitreous haze score and 3-line improvement from baseline in best corrected visual acuity [27]
Fluocinolone acetonide (intravitreal implant) Treatment of chronic non-infectious uveitis affecting the posterior segment of the eye Glucocorticoid 2000 2005 RCT 108 n/a 3 years Rate of recurrence of uveitis affecting the posterior segment of the study eye [28]
RCT 116 n/a 3 years Rate of recurrence of uveitis affecting the posterior segment of the study eye
Meloxicam For relief of the signs and symptoms of pauciarticular or polyarticular course juvenile rheumatoid arthritis in patients 2 years of age or older NSAID 2002 2005 RCT n/a ≥2 years 12 weeks ACR Pediatric 30 [29]
RCT n/a ≥2 years 12 weeks ACR Pediatric 30
Rilonacept Treatment of cryopyrin-associated periodic syndromes Interleukin inhibitor 2004 2008 RCT 47 n/a 24 weeks CAPS symptom score [30]
Adalimumab Treatment of juvenile idiopathic arthritis Tumor necrosis factor-alpha inhibitor 2005 2008 RCT 171 4 – 17 years 32 weeks ACR Pediatric 30 [31]
OL 32 2 – ≤ 4 years or ≥ 4 years weighing < 15 kg 120 weeks Safety
Rituximab For the use in combination with glucocorticoids for the treatment of patients with Wegener’s granulomatosis and microscopic polyangiitis Selective immunosuppressant 2006 2011 RCT 197 ≥15 years 18 months Birmingham Vasculitis Activity Score for Granulomatosis with Polyangiitis [16]
Colchicine Treatment of familial Mediterranean fever Mitotic poison 2007 2009 Evidence for the efficacy was derived from the published literature. [32]
RCT 15 n/a n/a n/a  
RCT 22 n/a n/a n/a
Canakinumab Treatment of cryopyrin-associated periodic syndromes, in adults and children 4 years of age and older Interleukin inhibitor 2007 2009 RCT 31 9 – 74 years 8 weeks Minimal or better for physician’s assessment of disease activity, assessment of skin disease, and serum levels of C-Reactive Protein and Serum Amyloid A [19]
OL n/a 4 – 74 years   n/a
Canakinumab Treatment of active systemic juvenile idiopathic arthritis in patients aged 2 through 16 years Interleukin inhibitor 2008 2013 RCT 84 mean 8.5 years (2 – 20) 4 weeks Adapted ACR Pediatric 30 and absence of fever [19]
RCT 177 mean 8.5 years (2 – 20) n/a ACR Pediatric 30
Difluprednate (ophthalmic solution) Treatment of endogenous anterior uveitis Glucocorticoid 2008 2012 RCT 110 n/a 2 weeks Difference in anterior chamber cell grade [33]
RCT 90 n/a 2 weeks Difference in anterior chamber cell grade
Anakinra Treatment of neonatal-onset multisystem inflammatory disease Interleukin inhibitor 2010 2012 OL 43 0.7 – 46 years 60 months disease-specific Diary Symptom Sum Score [34]
Tocilizumab Treatment of active polyarticular juvenile idiopathic arthritis in patients 2 through 16 years of age Interleukin inhibitor 2012 2013 RCT 188 2 – 17 years 24 weeks JIA ACR 30 flare [20]
  1. ACR Pediatric 30 American College of Rheumatology Pediatric 30 response, CAPS Cryopyrin-associated periodic syndromes, JIA Juvenile idiopathic arthritis, JIA ACR 30 flare Juvenile idiopathic arthritis American College of Rheumatology 30 flare, JRA Juvenile rheumatoid arthritis, n/a not applicable or no information is provided in the FDA label, NSAID Nonsteroidal anti-inflammatory drugs, OL Open label, RCT Randomized controlled trial, uncontrolled, SJIA Systemic juvenile idiopathic arthritis