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] |