Generic name | Condition | Categorisation | EMA status | FDA status | RCT results available |
---|---|---|---|---|---|
Abarelix | Prostate cancer | Solid tumour oncology | – | A | No |
Alemtuzumab | Chronic lymphocytic leukaemia (CLL) | Haematological oncology | A | A | No |
Alglucosidase alfa | Pompe disease | Rare metabolic condition | A | A | No |
Alipogene tiparvovec | Familial lipoprotein lipase deficiency | Rare metabolic condition | A | – | No |
Anagrelide | Essential thrombocytopenia | Blood count | A | A* | No |
Argatroban | Heparin-induced thrombocytopenia | Blood count | – | A* | No |
Arsenic trioxide | Acute promyelocytic leukaemia | Haematological oncology | A | A | No |
Asparaginase Erwinia chrysanthemi | Acute lymphoblastic leukaemia (ALL) | Haematological oncology | – | A | No |
Bendamustine hydrochloride | Non-Hodgkin's lymphoma | Haematological oncology | – | A | Yes |
Betaine anhydrous | Homocystinuria | Rare metabolic condition | A | – | No |
Bexarotene | Cutaneous T-cell lymphoma (CTCL) | Solid tumour oncology | A | A | No |
Bortezomib | Multiple myeloma (MM) | Haematological oncology | A | A | No |
Bortezomib | Mantle cell lymphoma (MCL) | Haematological oncology | – | A | Yes |
Bosutinib | Chronic myeloid leukaemia (CML) | Haematological oncology | A | A | Yes |
Brentuximab vedotin | Hodgkin's lymphoma (HL) | Haematological oncology | A | A | No |
Brentuximab vedotin | Systemic anaplastic large cell lymphoma (sALCL) | Haematological oncology | A | A | No |
Busulfan | Haematopoietic progenitor cell transplantation (HPCT) | Haematological oncology | A | A | Yes |
Carfilzomib | MM | Haematological oncology | – | A | No |
Carglumic acid | Chronic hyperammonaemia | Rare metabolic condition | A | A | No |
Ceritinib | Non-small cell lung cancer | Solid tumour oncology | – | A | No |
Cetuximab | Colorectal cancer | Solid tumour oncology | A | A | No |
Cholic acid (Kolbam) | Inborn errors in primary bile acid synthesis | Rare metabolic condition | A | – | No |
Cholic acid (Orphacol) | Inborn errors in primary bile acid synthesis | Rare metabolic condition | A | – | No |
Cladribine | Hairy cell leukaemia | Haematological oncology | A | – | No |
Clofarabine | ALL | Haematological oncology | A | A | No |
Crizotinib | Non-small-cell lung cancer | Solid tumour oncology | – | A | No |
Dasatinib | CML | Haematological oncology | A | A | No |
Dasatinib | Philadelphia chromosome-positive ALL | Haematological oncology | A | A | No |
Defibrotide | Veno-occlusive disease | Poisoning | A | – | Yes |
Dexrazoxane hydrochloride | Anthracycline extravasation | Poisoning | A | – | No |
Ferric hexacyanoferrate (II) | Internal contamination with radioactive caesium or thallium | Poisoning | – | A | No |
Gefitinib | Non-small cell lung cancer (NSCLC) | Solid tumour oncology | – | A | No |
Gemtuzumab ozogamicin | Acute myeloid leukaemia (AML) | Haematological oncology | S | S | No |
Glucarpidase | Toxic plasma methotrexate concentrations | Poisoning | S | A | No |
Hydroxocobalamin | Treatment of cyanide poisoning | Poisoning | A | A | No |
Ibrutinib | Mantle cell lymphoma (MCL) | Haematological oncology | – | A | No |
Ibrutinib | CLL | Haematological oncology | – | A | No |
Imatinib mesylate | Chronic myeloid leukaemia (CML) | Haematological oncology | A | A | No |
Imatinib mesylate | Gastrointestinal stromal tumours (GIST) | Solid tumour oncology | A | A | No |
Imatinib mesylate | Myelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene re-arrangements | Haematological oncology | A | A | Yes |
Imatinib mesylate | Soft tissue sarcoma—Dermatofibrosarcoma protuberans (DFSP) | Solid tumour oncology | A | A | Yes |
Imatinib mesylate | Philadelphia chromosome-positive acute ALL | Haematological oncology | – | A | Yes |
Imatinib mesylate | Aggressive systemic mastocytosis (ASM) | Haematological oncology | S | A | Yes |
Imatinib mesylate | Advanced hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukaemia (CEL) with FIP1L1-PDGFR rearrangement | Haematological oncology | A | A | Yes |
Ixabepilone | Breast cancer | Solid tumour oncology | S | A | Yes |
Lomitapide mesylate | Familial hypercholesterolemia (HoFH) | Rare metabolic condition | A | A | No |
Metreleptin | Lipodystrophy due to leptin deficiency | Rare metabolic condition | – | A | No |
Nelarabine | T-cell acute lymphoblastic leukaemia/lymphoma (T-ALL/T-LBL) | Haematological oncology | A | A | No |
Nilotinib hydrochloride monohydrate | Chronic myeloid leukaemia (CML) | Haematological oncology | A | A | No |
Nitisinone | Hereditary tyrosinaemia | Rare metabolic condition | A | A | No |
Ofatumumab | CLL | Haematological oncology | A | A | No |
Omacetaxine mepesuccinate | CML | Haematological oncology | S | A | No |
Paclitaxel | Kaposi's sarcoma | Solid tumour oncology | A | A | Yes |
Pasireotide diaspartate | Cushing's disease | Rare metabolic condition | A | A | No |
Pentetate calcium trisodium | Internal contamination with plutonium, americium, or curium | Poisoning | – | A | No |
Pentetate zinc trisodium | Internal contamination with plutonium, americium, or curium | Poisoning | – | A | No |
Pomalidomide | Multiple myeloma (MM) | Haematological oncology | – | A | No |
Ponatinib hydrochloride | CML | Haematological oncology | A | A | No |
Ponatinib hydrochloride | Philadelphia chromosome-positive acute ALL | Haematological oncology | A | A | No |
Pralatrexate | Peripheral T-cell lymphoma (PTCL) | Haematological oncology | S | A | No |
Raxibacumab | Anthrax inhalation | Poisoning | – | A | No |
Romidepsin | Peripheral T-cell lymphoma (PTCL) | Haematological oncology | S | A | Yes |
Sodium ferric gluconate complex | Iron deficiency | Rare metabolic condition | – | A | No |
Sodium phenylbutyrate | Urea cycle disorders | Rare metabolic condition | A | A* | No |
Sunitinib malate | Renal cell carcinoma | Solid tumour oncology | – | A | No |
Taliglucerase alfa | Gaucher's disease | Rare metabolic condition | S | A | No |
Temoporfin | Head and neck cancer | Solid tumour oncology | A | – | No |
Temozolomide | Anaplastic astrocytoma | Solid tumour oncology | A | A | Yes |
Tocofersolan | Vitamin E deficiency due to cholestasis | Rare metabolic condition | A | – | No |
Tositumomab; iodine I 131 tositumomab | Non-Hodgkin's lymphoma | Haematological oncology | – | A | No |
Trabectedin | Soft tissue sarcoma | Solid tumour oncology | A | – | No |
Vismodegib | Basal cell carcinoma | Solid tumour oncology | A | A | No |
Vorinostat | CTCL | Haematological oncology | S | A | No |
Zinc | Wilson's disease (hepatolenticular degeneration) | Rare metabolic condition | A | A* | No |
A, approved; A*, approved prior to 1999; S, submitted but not approved.