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
Omeprazole-lansoprazoe with buffer Esophageal ulcers Effexus Pharmaceuticals, LLC;10901 Granada Lane, Suite 101;Leawood, KS, 66211 February 10, 2015
Recombinant monoclonal antibody to human serum amyloid P component AL amyloidosis Glaxo Group Limited, England d/b/a GlaxoSmithKline;980 Great West Road, Middlesex, TW8, 9GS; February 10, 2015
Bivalent anti-human myostatin adnectin-IgG1 Duchenne muscular dystrophy Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 February 10, 2015
Copanlisib Follicular lymphoma Bayer US LLC;100 Bayer Boulevard, P. O. Box 915;Hanover, New Jersey, 07981 February 5, 2015
Antinuclear antibody conjugated liposomal doxorubicin Ewing's sarcoma NanoSmart Pharmaceuticals, Inc.;27071 Cabot Road, Suite 104;Laguna Hills, California, 92653 February 3, 2015
Bortezomib Acute lymphoblastic leukemia Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 February 3, 2015
N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4-methylpiperazin-1yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide Non-small cell lung cancer Ignyta, Inc.;4545 Towne Centre Court;San Diego, California, 92121 February 3, 2015
N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4-methylpiperazin-1yl)-2-(tetrahydro-2H-pyran-4-ylamino)benzamide TrkA-positive, TrkB-positive, TrkC-positive ROS1-positive and ALK-positive non-small cell lung cancer Ignyta, Inc.;11095 Flintkote Avenue, Suite D;San Diego, CA, 92121 February 3, 2015
Alectinib Non-small cell lung cancer Hoffmann-La Roche, Inc. (c/o Genentech, Inc.);1 DNA Way, MS #355E;South San Francisco, California, 94080 January 27, 2015
Filgrastim Amyotrophic lateral sclerosis Neurovision Pharma GmbH;Emil-Geis Str. 4;Grunwald January 27, 2015
18Fluorine-N-3-Fluoropropyl-2beta-carbomethoxy-3beta-(4-iodophenyl) Nortropane Multiple system atrophy Advanced Imaging Projects, LLC;7947 Brookside Ct.;Lake Worth, Florida, 33467 January 26, 2015
Theranost 68Ga RGD Diagnostic for the clinical management of patients with tuberculosis Advanced Imaging Projects, LLC;FL ATlantic University Research Park, 3651 FAU Blvd;Boca Raton, Florida, 33431 January 26, 2015
(S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile Pemphigus vulgaris Almirall S.A.;Ronda General Mitre, 151;Barcelona January 26, 2015
Double stranded RNA which targets the mutated KRAS oncogene Pancreatic Cancer Silenseed Ltd;Goldyne Savad Institute of Gene Therapy, Jerusalem, 9112001; January 26, 2015
2-(3-(4-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)-1H-pyrazol-l-yl)-1-(1-(3-fluoro-2-(trifluoromethyl)isonicotinoyl)piperidin-4-yl)azetidin-3-yl)acetonitrile adipate Pancreatic Cancer Incyte Corporation;1801 Augustine Cut-Off;Wilmington, Delaware, 19803 January 26, 2015
8-(1-Hydroxy-ethyl)-2-methoxy-3-(4-methoxy-benzyloxy)-benzo[c]chromen-6-one Glioblastoma Diffusion Pharmaceuticals, Inc.;2020 Avon Court, Suite 4;Charlottesville, Virginia, 22902 January 21, 2015
Antroquinonol Pancreatic Cancer Golden Biotechnology Corp.;10F., No. 9, Ln 3, Tamsui Dist., New Taipei City 251; January 21, 2015
5-(4-Cyclopropyl-1H-imidazol-l-yl)-2-fluoro-N-(6-(4-isopropyl-4H-1,2,4-triazol-3-yl)pyridin-2-yl)-4-methylbenzamide Pulmonary Arterial Hypertension Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 January 15, 2015
2,5-dimethyl-3-[2-methyl-4-(methyloxy)phenyl]-N-[(1S)-1-(3-methyl-1,2,4-oxadiazol-5- Congenital adrenal hyperplasia Neurocrine Biosciences, Inc.;12780 El Camino Real;San Diego, California, 92130 January 15, 2015
Autologous CD34+ bone marrow derived stem cells transduced with a self-inactivating gammaretroviral vector encoding the human IL2RG (yc) Severe combined immune deficiency-X1 Boston Children's Hospital;Div. of Hematology/Oncology, Karp 08215;Boston, Massachusetts, 02115 January 15, 2015
Arimoclomol citrate Neimann-Pick disease, type C Orphazyme ApS;Ole Maaloes Vej 3, 2200 Copenhagen; January 13, 2015
Deutetrabenazine Tourette syndrome TEVA Pharmaceuticals;3366 North Torrey Pines Court, Suite 225;LaJolla, California, 92037 January 13, 2015
Anti-Beta 1 integrin monoclonal antibody Ovarian cancer OncoSynergy, Inc.;409 Illinois Street, Room 3044;San Francisco, CA, 94158-2509 January 13, 2015
N-[2,6-bis(1-methylethyl)phenyl]-N’-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt Cushing’s syndrome Millendo Therapeutics, Inc.;301 North Main St., Suite 100;Ann Arbor,, Michigan, 48104 January 7, 2015
Pentosan polysulfate sodium Mucopolysaccharidosis type VI Plexcera Therapeutics, LLC;4445 North Highway A1A, Suite 241;Vero Beach, Florida, 32963 January 5, 2015