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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 |
---|---|---|---|---|
Cholesterol 24S-hydroxylase inhibitor | Dravet Syndrome | Takeda Development Center Americas, Inc.;Takeda Parkway;Deerfield, Illinois, 60015 | November 29, 2017 | |
Cotsiranib | Bile duct cancer | Sirnaomics, Inc.;401 Professional Drive, Suite 280;Gaithersburg, Maryland, 20879 | November 29, 2017 | |
Eubacterial Spores, Purified Suspension, Encapsulated | Ulcerative colitis | Seres Therapeutics, Inc.;200 Sidney Street;Cambridge, Massachusetts, 02139 | November 27, 2017 | |
CD4-mediated activated regulatory T cells | Graft Versus Host Disease | Universitatsmedizin der Johannes Gutenberg-Universitat Mainz;1 Langenbeckstra�e;Mainz | November 27, 2017 | |
Povidone iodine | Endophthalmitis | Veloce BioPharma LLC;7008 Lafayette Avenue;Fort Washington, Pennsylvania, 19034 | November 27, 2017 | |
(1R,2R)-1,2-cyclohexane-diaminepyrophosphato-platinum(II) | Multiple myeloma | Phosplatin Therapeutics LLC;1350 6th Avenue, 3rd Floor;New York, New York, 10019 | November 22, 2017 | |
Synthetic PreImplantation Factor (H-Met-Val-Arg-Ile-Lys-Pro-Gly-Ser-Ala-Asn-Lys-Pro-Ser-Asp-Asp-OH | Autoimmune hepatitis | BioIncept, LLC;140 East 40th Street, Suite 11E;New York, New York, 10016 | November 22, 2017 | |
Eflornithine | Neuroblastoma | KC Pharma, LLC;Towers Office Building, 11300 U.S. 1;Palm Beach Gardens, Florida, 33408 | November 22, 2017 | |
Cholera toxin B subunit | Ulcerative colitis | Aliment Wellness LLC;9462 Brownsboro Road, Suite 340;Louisville, Kentucky, 40241 | November 16, 2017 | |
DaxibotulinumtoxinA | Cervical dystonia | Revance Therapeutics, Inc.;7555 Gateway Boulevard;Newark, California, 94560 | November 16, 2017 | |
Human IgG2 anti-monoclonal antibody directed against mucosal addressin cell adhesion molecule (MAdCAM) | Ulcerative colitis | Shire Human Genetic Therapies, Inc. (Shire);300 Shire Way;Lexington, Massachusetts, 02421 | November 16, 2017 | |
Adeno-associated virus type 2 encoding human aromatic L-amino acid decarboxylase | Aromatic amino acid decarboxylase deficiency | University of California San Francisco;Department of Neurosurgery, 1855 Folsom Street;San Francisco, California, 94103 | November 14, 2017 | |
1,4-diamino-2,3-dicyano-1,4-bis[2-aminophenylthio]butadiene | Non-traumatic subarachnoid haemorrhage | Edvince AB | November 8, 2017 | |
(3R,3aS,9R,9aS,9bS)-3-((dimethylamino)methyl)-9-hydroxy-6,9-dimethyl-3,3a,4,5,7,8,9,9a-octahydroazuleno[4,5-b]furan-2(9bH)-one fumarate | Glioblastoma | Accendatech USA Incorporated;3730 Kirby Drive, Suite 1200;Houston, Texas, 77098 | November 8, 2017 | |
Corticotropin-releasing factor receptor-1 antagonist containing an unfused thiazole ring | Congenital adrenal hyperplasia | Spruce Biosciences;548 Market Street, Suite 74598;San Francisco, California, 94104 | November 8, 2017 | |
Ubidecarenone | Pancreatic Cancer | Berg, LLC;500 Old Connecticut Path, Building B;Framingham, Massachusetts, 01701 | November 8, 2017 | |
1-[4-bromo-5-[1-ethyl-7-(methylamino)-2-oxo-1,2-dihydro-1,6-naphthyridin-3-yl]-2-fluorophenyl]-3-phenylurea | Gastrointestinal stromal tumours | Worldwide Clinical Trials Limited | November 8, 2017 | |
(ethane-1,2-diylbis(oxy)bis(ethane-2,1-diyl)bis(bis(thiophene-2-ylmethyl)carbamate) | Malignant melanoma stages IIB to IV | 7 Hills Pharma, LLC;8285 El Rio Street, Suite 130;Houston, Texas, 77054 | November 8, 2017 | |
(1’R,6’R)-3-(benzylamine)-6-hydroxy-3′-methyl-4-pentyl-6′-(prop-1-en-2-yl)-[1,1′-bi(cyclohexane)]-2′,3,6-triene-2,5-dione | Systemic scleroderma | Quintiles Ireland Limited | November 8, 2017 | |
(R)-troloxamide quinone | Amyotrophic lateral sclerosis | Edison Orphan Pharma BV | November 8, 2017 | |
VLA-4 and LFA-1 integrin activator | Malignant melanoma stages IIB to IV | 7 Hills Pharma, LLC;8285 El Rio Street, Suite 130;Houston, Texas, 77054 | November 8, 2017 | |
3-aminopyridine-2-carboxaldehyde thiosemicarbazone | Stage 1B to IV cervical cancer | National Cancer Institute, Div of Cancer Treatment & Diagnosis (DCTD);9609 Medical Center Drive, Room 5-W520, MSC 9740;Rockville, Maryland, 20850 | November 8, 2017 | |
4-amino-1-[(1S,4R,5S)-2-fluoro-4,5-dihydroxy-3-(hydroxymethyl)cyclopent-2-en-1-yl]pyrimidin-2-one | Pancreatic Cancer | Quintiles Ireland Limited | November 8, 2017 | |
Alanyl-glutamine dipeptide | Prevention of complications associated with peritoneal dialysis (PD) treatment in end stage renal disease (ESRD) | Zytoprotec GmbH;28/2 Mariannengasse;Vienna | November 6, 2017 | |
(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide | Hemoglobinuria | Achillion Pharmaceuticals, Inc;300 George Street;New Haven, Connecticut, 06511 | November 2, 2017 |