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
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
Eubacterial Spores, Purified Suspension, Encapsulated Ulcerative colitis Seres Therapeutics, Inc.;200 Sidney Street;Cambridge, Massachusetts, 02139 November 27, 2017
Eflornithine Neuroblastoma KC Pharma, LLC;Towers Office Building, 11300 U.S. 1;Palm Beach Gardens, Florida, 33408 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
(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
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
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
Ubidecarenone Pancreatic Cancer Berg, LLC;500 Old Connecticut Path, Building B;Framingham, Massachusetts, 01701 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
(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
1,4-diamino-2,3-dicyano-1,4-bis[2-aminophenylthio]butadiene Non-traumatic subarachnoid haemorrhage Edvince AB 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
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
(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
(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
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
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