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

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.