Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

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Country Product Indication Sponsor Date
Lutetium-177(3+),S2,S7-cyclo[N-{4,7,10-tricarboxymethyl-1,4,7,10-tetraaza-cyclododecan-1-yl-acetyl}-4-chloro-L-phenylalanyl-D-cysteinyl-4-[(4S)-2,6-dioxo-1,3-diazinane-4-carboxamido]-L-phenylalanyl-4-(carbamoylamino)-D-phenylalanyl-L-lysyl-L-threonyl-L-cysteinyl-D-tyrosinamide] Gastro-entero-pancreatic neuroendocrine tumours Ipsen Pharma October 14, 2016
Carbamazepine Metaphyseal chondrodysplasia Schmid type University of Newcastle upon Tyne October 14, 2016
Melatonin Smith-Magenis syndrome Therapicon Srl October 14, 2016
N-[(2S)-5-{[(1R,2S)-2-(4-fluorophenyl)cyclopropyl]amino}-1-(4-methylpiperazin-1-yl)-1-oxopentan-2-yl]-4-(1H-1,2,3-triazol-1-yl)benzamide, bis-tosylate salt Myelofibrosis Imago BioSciences Ltd. October 14, 2016
Mogamulizumab Cutaneous T-cell lymphoma Kyowa Kirin Limited October 14, 2016
Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Duchenne muscular dystrophy Pharma Gateway AB October 14, 2016
2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide Invasive aspergillosis F2G Ltd October 14, 2016
P-ethoxy growth factor receptor-bound protein 2 antisense oligonucleotide Acute myeloid leukaemia Clinical Network Services (UK) Ltd October 14, 2016
Synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker Paroxysmal nocturnal hemoglobinuria Ra Europe Limited October 14, 2016
Adeno-associated viral vector serotype 2/2 containing a gene encoding the channelrhodopsin-2 protein Retinitis pigmentosa Allergan Pharmaceuticals International Limited October 14, 2016
Radio-iodinated (131I) anti-CD45 murine monoclonal antibody Treatment in haematopoietic stem cell transplantation PharmaLex UK Services Limited October 14, 2016
Tadekinig alfa Hemophagocytic lymphohistiocytosis Coté Orphan Consulting UK Limited October 14, 2016
Adeno-associated viral vector serotype 5 containing the humanRLBP1gene Retinitis pigmentosa HORAMA SA October 14, 2016
Self-complementary adeno-associated viral vector serotype 9 containing theSGSHgene Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) Abeona Therapeutics Europe SL October 14, 2016
Venetoclax Multiple myeloma AbbVie Ltd October 14, 2016
Acebutolol hydrochloride Smith-Magenis syndrome Therapicon Srl October 14, 2016
Tetrofosmin Glioma ProActina October 14, 2016
Autologous mononuclear cells derived from human cord blood Neonatal encephalopathy BrainRepair UG (haftungsbeschränkt) October 14, 2016
Ubiquinol Primary coenzyme Q10deficiency syndrome Consorcio Centro de Investigación Biomédica en Red M.P. October 14, 2016
Autologous mononuclear cells derived from human cord blood Leukomalacia BrainRepair UG (haftungsbeschränkt) October 14, 2016
Venetoclax Diffuse Large B-cell Lymphoma AbbVie Ltd October 14, 2016
A non-covalent trimer of tumour necrosis factor fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragment format Soft tissue sarcoma Philogen S.p.A. October 14, 2016
Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 Diffuse Large B-cell Lymphoma Novartis Europharm Limited October 14, 2016
Xenon Ischaemia reperfusion injury associated with cardiac arrest Neuroprotexeon Ltd October 14, 2016
Crenolanib besylate Soft tissue sarcoma Arog Pharmaceuticals Europe Ltd October 14, 2016