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
Ac-Leu-Thr-Phe-c[(R)-2-amino-2-methyldec-9-enoic acid-Glu-Tyr-Trp-Ala-Gln-Leu-(S)-2-amino-2-methylhept-6-enoic acid]1-Ala-Ala-Ala-Ala-Ala-D-Ala-NH2 Acute myeloid leukemia Aileron Therapeutics, Inc.;81 Albany Street;Cambridge, Massachusetts, 02139 April 19, 2017
Clemizole Dravet Syndrome Epygenix Therapeutics, Inc.;140 East Ridgewood Avenue, Suite 415 South Tower;Paramus, New Jersey, 07652 April 19, 2017
Peptide inhibiting murine double minute 2 (MDM2) and murine double minute X (MDMX) Acute myeloid leukemia Aileron Therapeutics, Inc.;81 Albany Street;Cambridge, Massachusetts, 02139 April 19, 2017
Immune globulin intravenous (human) octagam 10% Dermatomyositis OCTAPHARMA USA, Inc.;121 River Street, 12th Floor;Hoboken, New Jersey, 07030 April 19, 2017
Itacitinib Graft Versus Host Disease Incyte Corporation;1801 Augustine Cut-Off;Wilmington, Delaware, 19803 April 17, 2017
Huperzine A Dravet Syndrome Biscayne Neurotherapeutics, Inc.;4770 Biscayne Boulevard, Suite 660;Miami, Florida, 33137 April 12, 2017
Redaporfin Bile duct cancer Luzitin, S.A.;Edificio Bluepharma, S Martinho do Bispo;Coimbra April 12, 2017
Merestinib Biliary tract cancer Eli Lilly and Company;Lilly Corporate Center;Indianapolis, Indiana, 46285 April 10, 2017
Levosimendan Amyotrophic lateral sclerosis Orion Corporation;Orionintie 1, 0FI-02200 ESPOO; April 10, 2017
Recombinant human club cell protein Bronchiolitis obliterans Therabron Therapeutics, Inc.;9430 Key West Avenue;Rockville, Maryland, 20850 April 10, 2017
Tasquinimod Multiple myeloma Active Biotech AB;Scheelev�gen 22, P.O. Box 724;Lund April 10, 2017
Tubimod Active tuberculosis Immunitor, Inc.;365-2906 West Broadway, Vancouver, BC, V6K 2G8; April 10, 2017
Bromodomain and Extra-Terminal (BET) Inhibitor Acute myeloid leukemia FORMA Therapeutics, Inc.;500 Arsenal Street, Suite 100;Watertown, Massachusetts, 02472 April 5, 2017
Valproic acid Diffuse Large B-cell Lymphoma Valcuria AB;Scheelev�gen 2;Lund March 30, 2017
3,4-diaminopyridine Congenital myasthenic syndrome (CMS) Jacobus Pharmaceutical Co., Inc.;37 Cleveland Lane;Princeton, New Jersey, 08540 March 30, 2017
4-pyridinylmethyl 3(4-chlorophenyl)adamantine carboxamide Bile duct cancer RedHill Biopharma Ltd.;21 Ha'arba'a Street;Tel Aviv-Yafo March 30, 2017
(+)-a-dihydrotetrabenazine Tourette syndrome Adeptio Pharmaceuticals, Ltd;22 Bedford Row, WC1R 4JS;London March 30, 2017
MRG-106 oligonucleotide inhibitor of microRNA miR-155-5p Mycosis fungoides miRagen Therapeutics, Inc.;6200 Lookout Road, Suite 100;Boulder, Colorado, 80301 March 30, 2017
Liposomal oxaliplatin Bile duct cancer KC Specialty Therapeutics, LLC;2002 West 39th Avenue;Kansas City, Arkansas, 66103 March 30, 2017
Generation 4 hydroxyl-terminated polyamidoamine dendrimer containing an ethylene diamine (EDA) core, amidoamine repate units, and 64 hydroxyl end groups X-linked adrenoleukodystrophy Orpheris, Inc.;303 Twin Dolphin Drive, Suite 600;Redwood City, California, 94065 March 22, 2017
Ganitumab Ewing sarcoma NantCell, Inc.;9920 Jefferson Boulevard;Culver City, California, 90232 March 21, 2017
Vedolizumab Graft Versus Host Disease Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 March 21, 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
Acetylleucine Niemann-Pick disease IntraBio Ltd March 20, 2017