World health dilemmas: Orphan and rare diseases, orphan drugs and orphan patients

Table 3 Examples of orphan-designated drug products with at least one marketing approval in the United States for a rare disease indication

Drug product name	Orphan indications
Alglucerase injection	Replacement therapy in Gaucher's disease
Alitretinoin	Acute promyelocytic leukemia
Alpha1-Proteinase Inhibitor	Cystic fibrosis
Ambrisentan	Idiopathic pulmonary fibrosis
4-Aminosalicylic acid	Crohn's disease
Amifostine	Chemoprotective agent in cancer
Anagrelide	Polycythemia vera
Anti-tac (human)	Prevention of acute graft-vs-host disease
Arsenic trioxide	Multiple myeloma, MDS, CML, CLL
Atovaquone	Toxoplasma gondii encephalitis
Azacitidine	Acute myeloid leukemia
Beractant	Newborn infants with pneumonia
Bosentan	Idiopathic pulmonary fibrosis
Busulfan	Primary brain malignancies
Calfactant	Acute respiratory distress syndrome
Canakinumab	Juvenile idiopathic arthritis
Capsaicin	Erythromelalgia
Cladribine	Non-Hodgkin's lymphoma, CLL, AML
Clofarabine	Acute myelogenous leukemia
Coagulation factor VIIa	Bleeding in Glanzmann thrombasthenia
Cysteamine hydrochloride	Huntington's disease
Cytarabine	Gliomas
Daunorubicin liposomal	Acute myeloid leukemia
Decitabine	Sickle cell anemia, CML, AML
Eculizumab	Dermatomyositis
Epoprostenol	Replacement of heparin in hemodialysis patients
Filgrastim	Myelodysplastic syndrome and AIDS
Fludarabine phosphate	Non-Hodgkins lymphoma
Heme arginate	Myelodysplastic syndromes
Idarubicin	AML in pediatrics, MDS and CML
Ifosfamide	Bone and soft tissue sarcomas
Iloprost solution for infusion	Heparin-associated thrombocytopenia
Indium111 pentetreotide	Neuroendocrine tumors
Interferon gamma-1b	Idiopathic pulmonary fibrosis
Lenalidomide	Mantle cell lymphoma and CLL
Levocarnitine	Pediatric cardiomyopathy
Mecasermin	Amyotrophic lateral sclerosis
Mecasermin rinfabate	Burns that require hospitalization
Melphalan	Cutaneous melanoma
Mesna	Inhibition of the urotoxic effects
Miglustat	Neurological manifestations
Mitomycin-C	Refractory glaucoma
Mycophenolate mofetil	Pemphigus vulgaris
Nilotinib	Gastrointestinal stromal tumors
Nitazoxanide	Intestinal amebiasis
Nitisinone	Alkaptonuria
Nitric oxide	Acute respiratory distress syndrome
Pentostatin	Cutaneous T-cell lymphoma and CLL
Porfimer sodium	Cholangiocarcinoma
Pralatrexate	Diffuse large B-cell lymphoma
Primaquine phosphate	Pneumocystis carinii pneumonia
Protein C concentrate	Replacement therapy in protein C deficiency
Procarbazine hydrochloride	Malignant glioma
Quinine sulfate	Non Plasmodium falciparum malaria
Rapamycin (mTOR) inhibitor	Tuberous sclerosis complex
Rifabutin	Mycobacterium avium disease
Riluzole	Huntington's disease
Sermorelin acetate	Induction of ovulation in women
Sodium phenylbutyrate	Sickling disorders
Sodium thiosulfate	Platinum-induced ototoxicity
Somatropin	Induction of ovulation in women with infertility
Succimer	Mercury toxicity and kidney stones
Synthetic human secretin	Diagnostic procedures in pancreatic carcinoma
Synthetic porcine secretin	Diagnostic procedures in pancreatic carcinoma
Temozolomide	Advanced metastatic melanoma.
Tetrabenazine	Moderate/severe tardive dyskinesia
Thalidomide	Graft vs host disease in BMT
Topotecan HCl liposomal	Gliomas
Tretinoin	Acute and chronic leukemia
Trimetrexate	Metastatic carcinomas
Vorinostat	Multiple myeloma and mesothelioma

ALL: Acute lymphoblastic leukaemia; AML: Acute myeloid leukaemia; CML: Chronic myeloid leukemia; CLL: Chronic lymphocytic leukaemia; MDS: Myelodysplastic syndrome; BMT: Bone marrow transplantation. Adapted from references[43-45].