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
Adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase Ornithine Transcarbamylase Deficiency Dr Julien Baruteau March 20, 2017
Avacopan C3 glomerulopathy ChemoCentryx, Inc.;850 Maude Avenue;Mountain View, California, 94043 March 20, 2017
Allogeneic ex-vivo-expanded umbilical cord blood-derived hematopoietic CD34+ progenitor cells and allogeneic non-expanded umbilical cord blood-derived hematopoietic mature myeloid and lymphoid cells Treatment in haematopoietic stem cell transplantation Regulatory Resources Group Ltd March 20, 2017
Adeno-associated viral vector serotype rh.10 expressing beta-galactosidase Gangliosidosis generalized GM1- type 1 LYSOGENE March 20, 2017
Antisense oligonucleotide targeting theUSH2Agene Retinitis pigmentosa ProQR Therapeutics IV BV March 20, 2017
Autologous adipose tissue-derived mesenchymal stem cells Buerger’s disease SPC GmbH March 20, 2017
Cannabidiol Lennox-Gastaut syndrome GW Research Ltd March 20, 2017
1-(2-chlorophenyl)-1-(S)-hydroxy-2-(S)-carbamoyloxy-propane West syndrome Bio-Pharm Solutions, Co., Ltd.;6F, C Bldg., Advanced Institute of Convergence Technology, 145 Gwanggyo-Ro, Yeongtong-Gu;Suwon March 20, 2017
Phosphoinositide 3-kinase gamma peptide Cystic fibrosis Kither Biotech s.r.l. March 20, 2017
Liposomal annamycin Acute myeloid leukemia Moleculin Biotech, Inc.;2575 West Bellfort Avenue, Suite 333;Houston, Texas, 77054 March 20, 2017
Inebilizumab Neuromyelitis optica AstraZeneca AB March 20, 2017
Cell-penetrating dominant-negative activating transcription factor 5 Glioma Sapience Therapeutics, Inc;500 Mamaroneck Avenue, Suite 320;Harrison, New York, 10528 March 20, 2017
Poly-cyclodextrin-bis-cysteine-PEG3400-camptothecin-conjugate Ovarian cancer Viadoc Business Solutions Limited March 20, 2017
Ketoconazole Granulosa cell tumours Grupo Español de Tumores Huérfanos e Infrecuentes (GETHI) March 20, 2017
N-(3-(4-(3-(diisobutylamino)propyl)piperazin-1-yl)propyl)-1H-benzo[d]imidazol-2-amine disulphate salt Progressive supranuclear palsy Alzprotect sas;70 rue du docteur Alexandre Yersin;Loos March 20, 2017
Megestrol acetate Granulosa cell tumours Grupo Español de Tumores Huérfanos e Infrecuentes (GETHI) March 20, 2017
(3’R,4’S,5’R)-N-[(3R,6S)-6-carbamoyltetrahydro-2H-pyran-3-yl]-6”-chloro-4′-(2-chloro-3-fluoropyridin-4-yl)-4,4-dimethyl-2”-oxo-1”,2”-dihydrodispiro[cyclohexane-1,2′-pyrrolidine-3′,3”-indole]-5′-carboxamide mono(4-methylbenzenesulfonate) monohydrate Soft tissue sarcoma Daiichi Sankyo Europe GmbH March 20, 2017
Mutated form of human acetylcholine receptor a1 extracellular domain subunit Myasthenia gravis Myasthenia gravis Toleranzia AB;Arvid Wallgrens backe 20;Gothenburg March 16, 2017
Synthetic, single stranded, fully phosphorothioated 2�-OMethyl-RNA and DNA mixmer oligonucleotide-based compound targeted against natural antisense transcripts (NATs) Dravet Syndrome OPKO Ireland Global Holdings Ltd.;10 Market Street, Suite 721;Camana Bay March 16, 2017
Talabostat Pancreatic Cancer BioXcel Corporation;780 East Main Street, Suite 2;Branford, Connecticut, 06405 March 16, 2017
Pentetrazol Narcolepsy Balance Therapeutics, Inc.;1250 Bayhill Drive, Suite 125;San Bruno, California, 94066 March 16, 2017
Recombinant human placental growth factor Severe preeclampsia Aggamin, LLC;3960 Broadway, Suite 330B;New York, New York, 10032 March 16, 2017
Synthetic, single stranded, fully phosphorothioated 2’-OMethyl-RNA and DNA mixmer oligonucleotide-based compound targeted against natural antisense transcripts (NATs) Dravet Syndrome OPKO Ireland Global Holdings Ltd.;10 Market Street, Suite 721;Camana Bay March 16, 2017
Activated natural killer[aNK]) Merkel cell cancer NantKwest, Inc.;9920 Jefferson Boulevard;Culver City, California, 90232 March 15, 2017
Fixed dose combination of methimazole and liothyronine Brain metastases in patients with primary lung cancer Musli Thyropeutics Ltd.;6 Hagefen Street, P. O. Box 529;Zur Moshe March 6, 2017