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

Table 5 List of drugs approved for rare diseases in Europe

Rare diseasecategory	Drug product name (Indication)
Leukaemias, lymphomas and related diseases	Histamine dihydrochloride and Decitabine (AML)
	Ofatumumab (CLL)
	Nilotinib (CML)
	Mercaptopurine and Clofarabine (ALL)
	Cladribine (Hairy cell leukaemia)
	Ponatinib (Philadelphia chromosome positive ALL and CML)
	Dasatinib (CML and AML)
	Azacitidine (Myelodysplastic syndromes, CML, AML)
	Bosutinib (Philadelphia chromosome positive CML)
	Nelarabine (T-cell ALL and lymphoma)
	Brentuximab vedotin (Hodgkin lymphoma and anaplastic large cell lymphoma)
	Ruxolitinib (Primary and other myelofibrosis cases)
	Plerixafor (Lymphoma and multiple myeloma)
Carcinomas and related diseases	Sorafenib tosylate (Hepatocellular carcinoma, renal cell carcinoma)
	Mitotane (Adrenal cortical carcinoma)
	Mifamurtide (Osteosarcoma)
	Temsirolimus (Renal cell carcinoma and mantle cell lymphoma)
	Trabectedin (Soft tissue sarcoma, liposarcoma, ovarian cancer)
	5-Aminole-vulinic-acid hydrochloride (Malignant glioma)
	Thalidomide and Lenalidomide (Multiple myeloma)
Chelating drugs and haemoglo- binopathy related diseases	Deferoxamine, Deferiprone and Deferasirox (Iron overload in beta thalassaemia)
	Dexrazoxane (Anthracycline extravasation)
	Hydroxycarbamide (Sickle Cell Syndrome)
	Zinc acetate dehydrate (Wilson's disease)
	Eculizumab (Proxysmal nocturnal haemoglobinuria and haemolytic uraemia)
Pulmonary hypertension	Bosentan monohydrate (Pulmonary arterial and systemic sclerosis)
	Iloprost (Primary pulmonary hypertension)
	Ambrisentan and Sildanafil citrate (Pulmonary arterial hypertension)
Cystic fibrosis	Mannitol, Aztreonam and Ivacaftor (Cystic fibrosis)
	Tobramycin (Pseudomonas aeruginosa in Cystic Fibrosis)
Enzymes used as drugs	Velaglucerase alpha (Type 1 Gaucher disease)
	Alpha-glucosidase (Pompe disease)
	Galsulfase (Mucopolysaccharidosis VI)
	Idursulfase (Hunter syndrome)
	Proteolytic enzymes enriched in bromelain (Burns)
Drugs used in other rare conditions	Carglumic acid (Hyperammonaemia due to n-acetylglutamate synthase deficiency)
	Betaine anhydrous (Homocystinuria)
	Stiripentol (Severe myoclonic epilepsy in infancy)
	Pirfenidone (Idiopathic Pulmonary Fibrosis)
	Icatibant acetate (Hereditary angioedema)
	Amifampridine (Lambert-Eaton myasthenic syndrome)
	Alipogene tiparvovec (Familial lipoprotein lipase deficiency)
	Mecasermin (Growth failure in primary insulin-like growth factor 1 deficiency)
	Rufinamide (Lennox Gastaut syndrome)
	Sapropterin dihydrochloride (Phenylketonuria and tetrahydropterin deficiency)
	Romiplostim (Immune thrombocytopenic purpura)
	Nitisinone (Hereditary tyrosinemia type 1)
	Ibuprofen (Patent ductus arteriosus)
	Caffeine citrate (Primary apnea)
	Hydrocortisone (Adrenal insufficiency)
	Zicotide (Chronic pain)
	Teduglutide (Short Bowel Syndrome)
	Pasireotide (Cushing's disease)
	Thiotepa (Haematopoietic progenitor cell transplantation)
	Everolimus (Tuberous sclerosis complex)
	Tafamidis (Transthyretin amyloidosis)
	Anargelide hydrochloride (Essential thrombocythaemia)
	Miglustat (Type 1 Gaucher disease and Niemann-Pick type C disease)

ALL: Acute lymphoblastic leukaemia; AML: Acute myeloid leukaemia; CML: Chronic myeloid leukemia; CLL: Chronic lymphocytic leukaemia. Adapted from ref[46].