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Table 1 Drug class and indications of designated anti-rheumatic orphan drugs

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

Drug class Compound Designated indication according to FDA database
Biologic   
Chimeric monoclonal antibody Infliximab Treatment of juvenile rheumatoid arthritis.
Treatment of giant cell arteritis.
Treatment of chronic sarcoidosis.
  Rituximab For the use in combination with glucocorticoids for the treatment of patients with Wegener’s granulomatosis (WG) and microscopic polyangiitis (MPA).
Human monoclonal antibody Adalimumab Treatment of juvenile rheumatoid arthritis.
  Canakinumab Treatment of pediatric (age 16 and under) juvenile rheumatoid arthritis.
Treatment of cryopyrin-associated periodic syndromes.
Treatment of Tumor Necrosis Factor-receptor associated periodic syndrome (TRAPS).
Treatment of hyperimmunoglobulinemia D and periodic fever syndrome.
Treatment of familial mediterranean fever.
  Golimumab Treatment of chronic sarcoidosis.
Treatment of sarcoidosis.
  Secukinumab Adjunctive treatment of chronic non-infectious uveitis requiring systemic immunosuppression.
  Ustekinumab Treatment of chronic sarcoidosis.
Humanized monoclonal antibody Eculizumab Treatment of dermatomyositis.
  Gevokizumab Treatment of Behçet disease.
Treatment of non-infectious intermediate, posterior or pan uveitis, or chronic non-infectious anterior uveitis.
  Humanized monoclonal antibody to CD40L (IDEC-131) Treatment of systemic lupus erythematosus.
  Humanized, afucosylated IgG1 kappa monoclonal antibody Treatment of scleroderma.
  Mepolizumab Treatment of Churg-Strauss Syndrome.
  Recombinant humanized monoclonal antibody 5c8 Treatment of systemic lupus erythematosus.
  Tocilizumab Treatment of pediatric patients (age 16 years and younger) with polyarticular-course juvenile idiopathic arthritis.
Unspecified monoclonal antibody Human anti- Tumor Necrosis Factor alpha monoclonal antibody Treatment of uveitis of the posterior segment of non-infectious etiology, and uveitis of the anterior segment of non-infectious etiology and refractory to conventional therapy.
  Monoclonal antibody for immunization against lupus nephritis Treatment of lupus nephritis.
Fusion protein 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.
Treatment of Wegener’s granulomatosis.
  Rilonacept Treatment of CIAS1-associated periodic syndromes.
Treatment of familial Mediterranean fever.
Other Anakinra Treatment of cryopyrin-associated periodic syndromes.
  Hanferon Treatment of Behçet disease.
  Interferon beta-1a Treatment of juvenile rheumatoid arthritis.
  Interleukin-1 receptor antagonist, human recombinant Treatment of juvenile rheumatoid arthritis.
  pVGI.1 (VEGF2) Treatment of thromboangiitis obliterans.
Small molecule 8-methoxsalen For use in conjunction with the UVAR photopheresis to treat diffuse systemic sclerosis.
  Apremilast Treatment of Behçet disease.
  Bindarit Treatment of lupus nephritis.
  Bromhexine Treatment of mild to moderate keratoconjunctivitis sicca in patients with Sjögren's syndrome.
  Clindamycin hydrochloride Treatment of sarcoidosis.
  Colchicine Treatment of familial Mediterranean fever.
Treatment of Behçet Syndrome.
  Cyclosporine (ophthalmic) Treatment of severe keratoconjunctivitis sicca associated with Sjogren’s syndrome.
  Dehydroepiandrosterone Treatment of systemic lupus erythematosus (SLE) and the reduction in the use of steroids in steroid-dependent SLE patients.
  Dexamethasone (intravitreal implant) Treatment of non-infectious ocular inflammation of the posterior segment in patients with intermediate, posterior, and panuveitis.
  Difluprednate (ophthalmic solution) Treatment of endogenous and traumatic anterior uveitis and panuveitis.
  Dimethyl sulfoxide Treatment of cutaneous manifestations of scleroderma.
  Fluocinolone acetonide (intravitreal implant) Treatment uveitis involving the posterior segment of the eye.
  Gammalinolenic acid Treatment of juvenile rheumatoid arthritis.
  Guanethidine monosulfate Treatment of moderate to severe reflex sympathetic dystrophy and causalgia.
  Gusperimus trihydrochloride Treatment of Wegener’s granulomatosis.
  Meloxicam Treatment of juvenile rheumatoid arthritis.
  Methotrexate Treatment of juvenile rheumatoid arthritis.
  Minocycline hydrochloride Treatment of sarcoidosis.
  Nabumetone Treatment of pediatric juvenile rheumatoid arthritis.
  N-acetyl-procainamide Prevention of life-threatening ventricular arrhythmias in patients with documented procainamide-induced lupus.
  Nitric oxide Diagnosis of sarcoidosis.
  Pentoxifylline Treatment of Behçet disease.
  Pilocarpine HCl Treatment of xerostomia and keratoconjunctivitis sicca in Sjögren's syndrome patients.
  Rofecoxib Treatment of juvenile rheumatoid arthritis.
  Siponimod Treatment of polymyositis.
  Sirolimus Treatment of chronic/refractory anterior noninfectious uveitis, noninfectious intermediate uveitis, noninfectious panuveitis and non-infectious, uveitis affecting the posterior of the eye (NICUPS).
  Thymopentin Treatment of sarcoidosis.
Other Abetimus Treatment of lupus nephritis.
  Allogeneic ex-vivo expanded placental adherent stromal cells Treatment of thromboangiitis obliterans (Buerger’s disease).
  Bone marrow-derived mononuclear cells Treatment of thromboangiitis obliterans (Buerger’s disease).
  Cyclo {{(E,Z)-(2S, 3R, 4R)-3-hydroxy-4-methyl-2-(methylamino) nona-6,8-dienoyl}-L-2-aminobytyrl-N-methyl-glycyl-N-methyl-L-leucyl-L-valyl-N-methyl-L-leucyl-L-alanyl-D-alanyl-N-methyl-L-leucyl-N-methyl-L-leucyl-N-methyl-L-valyl} Treatment and chronic control of non-infectious posterior, intermediate and pan-uveitis.
  Human gammaglobulin Treatment for juvenile rheumatoid arthritis.
Treatment of idiopathic inflammatory myopathies.
  Immune globulin intravenous (IVIG) Treatment of juvenile rheumatoid arthritis.
Treatment of polymyositis/dermatomyositis.
  Kre-Celazine (Oral Buffered Creatine-Cetylated Fatty Acid Compound) Treatment of juvenile rheumatoid arthritis joint and related tissue inflammation in the pediatric population.
  Lactobacillus brevis CD2 Treatment Behçet disease.
  L-pyr-L-glu-L-gln-L-leu-L-glu-L-arg-L-ala-L-leu-L-asn-L-ser-L-ser Treatment of neuropathic pain in patients with sarcoidosis.
  Natural human lymphoblastoid interferon-alpha Treatment of Behçet disease.
  Peptide 144 (TGF beta-1-inhibitor) Treatment of localized scleroderma.
  Purified type II collagen Treatment of juvenile rheumatoid arthritis.
Unknown AI-RSA Treatment of autoimmune uveitis.
  Interleukin-1 Trap Treatment of Still’s disease including juvenile rheumatoid arthritis and adult-onset Still’s disease.