Treatments

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

You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.

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Country Product Indication Sponsor Date
Adeno-associated viral vector serotype rh.10 expressing beta-galactosidase Gangliosidosis generalized GM1- type 1 LYSOGENE 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
Autologous adipose tissue-derived mesenchymal stem cells Buerger’s disease SPC GmbH March 20, 2017
Antisense oligonucleotide targeting theUSH2Agene Retinitis pigmentosa ProQR Therapeutics IV BV 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
Cannabidiol Lennox-Gastaut syndrome GW Research Ltd 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
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
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
Ketoconazole 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
Megestrol acetate Granulosa cell tumours Grupo Español de Tumores Huérfanos e Infrecuentes (GETHI) March 20, 2017
Talabostat Stages IIb through IV melanoma BioXcel Corporation;780 East Main Street, Suite 2;Branford, Connecticut, 06405 March 20, 2017
Adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene Fabry disease Freeline Therapeutics Ltd March 20, 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
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
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