Table 2

Drugs submitted to the EMA and the FDA containing only uncontrolled clinical studies

Generic nameConditionCategorisationEMA statusFDA statusRCT results available
AbarelixProstate cancerSolid tumour oncologyANo
AlemtuzumabChronic lymphocytic leukaemia (CLL)Haematological oncologyAANo
Alglucosidase alfaPompe diseaseRare metabolic conditionAANo
Alipogene tiparvovecFamilial lipoprotein lipase deficiencyRare metabolic conditionANo
AnagrelideEssential thrombocytopeniaBlood countAA*No
ArgatrobanHeparin-induced thrombocytopeniaBlood countA*No
Arsenic trioxideAcute promyelocytic leukaemiaHaematological oncologyAANo
Asparaginase Erwinia chrysanthemiAcute lymphoblastic leukaemia (ALL)Haematological oncologyANo
Bendamustine hydrochlorideNon-Hodgkin's lymphomaHaematological oncologyAYes
Betaine anhydrousHomocystinuriaRare metabolic conditionANo
BexaroteneCutaneous T-cell lymphoma (CTCL)Solid tumour oncologyAANo
BortezomibMultiple myeloma (MM)Haematological oncologyAANo
BortezomibMantle cell lymphoma (MCL)Haematological oncologyAYes
BosutinibChronic myeloid leukaemia (CML)Haematological oncologyAAYes
Brentuximab vedotinHodgkin's lymphoma (HL)Haematological oncologyAANo
Brentuximab vedotinSystemic anaplastic large cell lymphoma (sALCL)Haematological oncologyAANo
BusulfanHaematopoietic progenitor cell transplantation (HPCT)Haematological oncologyAAYes
CarfilzomibMMHaematological oncologyANo
Carglumic acidChronic hyperammonaemiaRare metabolic conditionAANo
CeritinibNon-small cell lung cancerSolid tumour oncologyANo
CetuximabColorectal cancerSolid tumour oncologyAANo
Cholic acid (Kolbam)Inborn errors in primary bile acid synthesisRare metabolic conditionANo
Cholic acid (Orphacol)Inborn errors in primary bile acid synthesisRare metabolic conditionANo
CladribineHairy cell leukaemiaHaematological oncologyANo
ClofarabineALLHaematological oncologyAANo
CrizotinibNon-small-cell lung cancerSolid tumour oncologyANo
DasatinibCMLHaematological oncologyAANo
DasatinibPhiladelphia chromosome-positive ALLHaematological oncologyAANo
DefibrotideVeno-occlusive diseasePoisoningAYes
Dexrazoxane hydrochlorideAnthracycline extravasationPoisoningANo
Ferric hexacyanoferrate (II)Internal contamination with radioactive caesium or thalliumPoisoningANo
GefitinibNon-small cell lung cancer (NSCLC)Solid tumour oncologyANo
Gemtuzumab ozogamicinAcute myeloid leukaemia (AML)Haematological oncologySSNo
GlucarpidaseToxic plasma methotrexate concentrationsPoisoningSANo
HydroxocobalaminTreatment of cyanide poisoningPoisoningAANo
IbrutinibMantle cell lymphoma (MCL)Haematological oncologyANo
IbrutinibCLLHaematological oncologyANo
Imatinib mesylateChronic myeloid leukaemia (CML)Haematological oncologyAANo
Imatinib mesylateGastrointestinal stromal tumours (GIST)Solid tumour oncologyAANo
Imatinib mesylateMyelodysplastic/myeloproliferative diseases (MDS/MPD) associated with platelet-derived growth factor receptor (PDGFR) gene re-arrangementsHaematological oncologyAAYes
Imatinib mesylateSoft tissue sarcoma—Dermatofibrosarcoma protuberans (DFSP)Solid tumour oncologyAAYes
Imatinib mesylatePhiladelphia chromosome-positive acute ALLHaematological oncologyAYes
Imatinib mesylateAggressive systemic mastocytosis (ASM)Haematological oncologySAYes
Imatinib mesylateAdvanced hypereosinophilic syndrome (HES) and/or chronic eosinophilic leukaemia (CEL) with FIP1L1-PDGFR rearrangementHaematological oncologyAAYes
IxabepiloneBreast cancerSolid tumour oncologySAYes
Lomitapide mesylateFamilial hypercholesterolemia (HoFH)Rare metabolic conditionAANo
MetreleptinLipodystrophy due to leptin deficiencyRare metabolic conditionANo
NelarabineT-cell acute lymphoblastic leukaemia/lymphoma (T-ALL/T-LBL)Haematological oncologyAANo
Nilotinib hydrochloride monohydrateChronic myeloid leukaemia (CML)Haematological oncologyAANo
NitisinoneHereditary tyrosinaemiaRare metabolic conditionAANo
OfatumumabCLLHaematological oncologyAANo
Omacetaxine mepesuccinateCMLHaematological oncologySANo
PaclitaxelKaposi's sarcomaSolid tumour oncologyAAYes
Pasireotide diaspartateCushing's diseaseRare metabolic conditionAANo
Pentetate calcium trisodiumInternal contamination with plutonium, americium, or curiumPoisoningANo
Pentetate zinc trisodiumInternal contamination with plutonium, americium, or curiumPoisoningANo
PomalidomideMultiple myeloma (MM)Haematological oncologyANo
Ponatinib hydrochlorideCMLHaematological oncologyAANo
Ponatinib hydrochloridePhiladelphia chromosome-positive acute ALLHaematological oncologyAANo
PralatrexatePeripheral T-cell lymphoma (PTCL)Haematological oncologySANo
RaxibacumabAnthrax inhalationPoisoningANo
RomidepsinPeripheral T-cell lymphoma (PTCL)Haematological oncologySAYes
Sodium ferric gluconate complexIron deficiencyRare metabolic conditionANo
Sodium phenylbutyrateUrea cycle disordersRare metabolic conditionAA*No
Sunitinib malateRenal cell carcinomaSolid tumour oncologyANo
Taliglucerase alfaGaucher's diseaseRare metabolic conditionSANo
TemoporfinHead and neck cancerSolid tumour oncologyANo
TemozolomideAnaplastic astrocytomaSolid tumour oncologyAAYes
TocofersolanVitamin E deficiency due to cholestasisRare metabolic conditionANo
Tositumomab; iodine I 131 tositumomabNon-Hodgkin's lymphomaHaematological oncologyANo
TrabectedinSoft tissue sarcomaSolid tumour oncologyANo
VismodegibBasal cell carcinomaSolid tumour oncologyAANo
VorinostatCTCLHaematological oncologySANo
ZincWilson's disease (hepatolenticular degeneration)Rare metabolic conditionAA*No
  • A, approved; A*, approved prior to 1999; S, submitted but not approved.