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
NiCord (cord blood-derived exvivo expanded CD34+ cells and the non-expanded cell fraction of the same cord blood unit) Chronic myeloid leukemia Gamida Cell Ltd.;P. O. Box 34670;Jerusalem November 9, 2016
Rituximab Rasmussen's encephalitis Keck Graduate Institute of Applied Life Sciences;535 Watson Drive;Claremont, California, 91711 November 9, 2016
Recombinant growth hormone fused to hyFc Growth hormone deficiency Genexine, Inc.;700 Daewangpangyo-ro, Korea Bio Pk Bldg B, Bundang-gu;Gyeonggi-do November 9, 2016
Autologous stromal vascular cell fraction from adipose tissue (central facitily processed) Systemic scleroderma Cytori Therapuetics, Inc.;3020 Callan Road;San Diego, California, 92121 November 9, 2016
6-(4-(4-(2,3-dichlorophenyl)-piperazin-1-yl)-butoxy)-2H-benzo[b][1,4]oxazin-3(4H)-one hydrochloride Pulmonary Arterial Hypertension Reviva Pharmaceuticals, Inc.;3900 Freedom Circle, Suite 101;Santa Clara, California, 95054 November 9, 2016
Sargramostim Acute Radiation Syndrome Acute radiation syndrome Sanofi Genzyme;500 Kendall Street;Cambridge, Massachusetts, 02142 November 9, 2016
Riboflavin-vitamin E-TPGS topical solution Keratoconus Institute of Refractive & Ophthalmic Surgery;Via Andrea D'Isernia 31,;Napoli November 8, 2016
Aganirsen Prevention of corneal graft rejection Gene Signal International SA;Route Cantonale;Lausanne November 7, 2016
2-[4-[[(2R)-2-ethoxy-3-[4-(trifluoromethyl)phenoxy]propyl]thio]-2-methylphenoxy]acetic acid (1:1) lysine dihydrate Primary biliary cirrhosis CymaBay Therapeutics;7999 Gateway Boulevard, Suite 130;Newark, California, 94560 November 7, 2016
6′-(R)-Methyl-5-O-(5-amino-5,6-dideoxy-a-L-talofuranosyl)- paromamine sulfate Rett syndrome Eloxx Pharmaceuticals, Ltd.;Weizmann Science Park, 7 Golda Meir Street;Ness Ziona November 3, 2016
2-amino-3-[3-[[4-(2-pyridyloxymethyl)phenyl]methyl]isoxazol-5-yl]pyridin-1-ium-1-yl]methyl hydrogen phosphate Coccidioidomycosis Amplyx Pharmaceuticals, Inc.;12730 High Bluff Drive, Suite 160;San Diego, California, 92130 November 3, 2016
Veliparib Non-small cell lung cancer AbbVie Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 November 3, 2016
Ruxolitinib Graft Versus Host Disease Incyte Corporation;1801 Augustine Cut-Off;Wilmington, Delaware, 19803 November 3, 2016
Melatonin Smith-magenis syndrome in combination with a beta-blocker Therapicon SrL;21 Via Malachia Marchesi de Taddei;Milano November 3, 2016
H-(4-amino-3-iodo)-D-Phe-c[Cys-(3-iodo)-Tyr-D-Trp-Lys-Val-Cys]-Thr-NH2 Acromegaly Amryt Research Ltd.;18 Fitzwilliam Place;Dublin November 3, 2016
2-amino-3-[3-[[4-(2-pyridyloxymethyl)phenyl]methyl]isoxazol-5-yl]pyridin-1-ium-1-yl]methyl hydrogen phosphate Invasive rare mold infections caused by scedosporium spp., fusarium spp., and mucorales fungi (including mucor spp., and rhizopus spp.) Amplyx Pharmaceuticals, Inc.;12730 High Bluff Drive, Suite 160;San Diego, California, 92130 November 2, 2016
2-amino-3-[3-[[4-(2-pyridyloxymethyl)phenyl]methyl]isoxazol-5-yl]pyridin-1-ium-1-yl]methyl hydrogen phosphate Invasive aspergillosis Amplyx Pharmaceuticals, Inc.;12730 High Bluff Drive, Suite 160;San Diego, California, 92130 October 26, 2016
Napabucasin Pancreatic cancer Boston Biomedical, Inc.;640 Memorial Drive;Cambridge, Massachusetts, 02139 October 25, 2016
Sofosbuvir Pediatric chronic hepatitis C virus infection Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 October 25, 2016
Rapamycin Pulmonary arterial hypertention Lam Therapeutics;530 Old Whitfield Street;Guilford, Connecticut, 06437 October 20, 2016
Fixed dose combination formulation of macitentan and tadalafil Pulmonary Arterial Hypertension Actelion Clinical Research, Inc.;1820 Chapel Avenue West, Suite 300;Cherry Hill, New Jersey, 08002 October 19, 2016
Partial agonist at the opioid receptor-like 1 receptor with amino acid sequence Ac-Arg-Tyr-Tyr-Arg-Trp-Lys-Lys-Lys-Lys-Lys-Lys-Lys-NH2 Pulmonary Arterial Hypertension Serodus ASA;Gaustadall�en 21;Oslo October 19, 2016
2-amino-3-[3-[[4-(2-pyridyloxymethyl)phenyl]methyl]isoxazol-5-yl]pyridin-1-ium-1-yl]methyl hydrogen phosphate Invasive candidiasis Amplyx Pharmaceuticals, Inc.;12730 High Bluff Drive, Suite 160;San Diego, California, 92130 October 19, 2016
6-(R)-Methyl-5-O-(5-amino-5,6-dideoxy-a-L-talofuranosyl)- paromamine sulfate Hurler syndrome Eloxx Pharmaceuticals, Ltd.;Weizmann Science Park, 7 Golda Meir Street;Ness Ziona October 18, 2016
(E)-(6-((N-methyl-((3-methylbenzofuran-2-yl)methyl)amino)-3-oxoprop-1-en-1-yl)-2-oxo-3,4-dihydro-1,8-naphthyridin-1(2H)-yl)methyl phosphate, bis ethanolamine salt Osteomyelitis Voisin Consulting S.A.R.L. October 14, 2016