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
Ondansetron inhalation powder Prevention of chemotherapy-induced nausea and vomiting due to highly emetogenic chemotherapy in pediatric patients (0 through 16 years of age) Luxena Pharmaceuticals, Inc.;1400 Coleman Avenue, Suite D27;Santa Clara, California, 95050 October 14, 2015
Human soluble receptor-Fc fusion protein that targets human activin A Ovarian cancer Atara Biotherapeutics;4360 Park Terrace Drive, Suite 100;Westlake Village, California, 91361 October 14, 2015
(6aR, 10aR)-3-(1’,1’-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Cystic fibrosis Corbus Pharmaceuticals, Inc.;100 River Ridge Drive, Suite 103;Norwood, Massachusetts, 02062 October 13, 2015
(6aR, 10aR)-3-(1�,1�-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Cystic fibrosis Corbus Pharmaceuticals, Inc.;100 River Ridge Drive, Suite 103;Norwood, Massachusetts, 02062 October 13, 2015
Ovine-specific immunoglobulin (Fab) fragments raised againstVipera berusvenom Snakebite envenomation MicroPharm Limited October 9, 2015
Sirolimus Tuberous sclerosis Desitin Arzneimittel GmbH October 9, 2015
3-pentylbenzeneacetic acid sodium salt Idiopathic pulmonary fibrosis ProMetic Pharma SMT Limited October 9, 2015
2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione Systemic scleroderma GenKyoTex Innovation S.A.S October 9, 2015
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor small lymphocytic lymphoma Kite Pharma EU B.V. October 9, 2015
N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide Duchenne muscular dystrophy FGK Representative Service GmbH October 9, 2015
Nimodipine Aneurysmal subarachnoid haemorrhage Dr Stefan Blesse October 9, 2015
Ataluren Aniridia PTC Therapeutics International Limited October 9, 2015
Synthetic hepcidin Beta thalassaemia intermedia and major Cogas Pharma Limited October 9, 2015
A highly purified formulation ofStaphylococcus aureusprotein A Immune thrombocytopenia Coté Orphan Consulting UK Limited October 9, 2015
Recombinant adeno-associated viral vector containing humanCNGA3gene Achromatopsia TMC Pharma Services Ltd October 9, 2015
2-chloro-N6-(3-iodobenzyl)adenosine-5′-N-methyluronamide Hepatocellular Carcinoma PBS Regulatory Consulting Group Limited October 9, 2015
Three chimeric human/murine monoclonal antibodies against the Ebola (Zaire) surface glycoprotein Ebola Dr Stefan Blesse October 9, 2015
Recombinant human interleukin-3 truncated diphtheria toxin fusion protein Acute myeloid leukaemia TMC Pharma Services Ltd October 9, 2015
Autologous human peripheral blood Vdelta1+ T lymphocytes activated in vitro by cytokine and monoclonal antibody treatment small lymphocytic lymphoma Lymphact - Lymphocyte Activation Technologies S.A. October 9, 2015
Synthetic peptide L-cysteine, L-cysteinylglycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysyl-L-prolyl-L-tryptophyl-L-tyrosyl-, cyclic (1.fwdarw.17)-disulfide Primary graft dysfunction following lung transplantation Apeptico Forschung und Entwicklung GmbH October 9, 2015
Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein Hereditary angioedema Shire Pharmaceuticals Ireland Limited October 9, 2015
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor Mantle cell lymphoma Kite Pharma EU B.V. October 9, 2015
Betamethasone Ataxia-Telangiectasia Grace Therapeutics, LLC;675 U.S. 1;North Brunswick Township, New Jersey, 08902 October 7, 2015
Mixture of 2 anti-rabies humanized monoclonal immunoglobulin (Ig)G1k antibodies Rabies Synermore Biologics Co., Ltd;6F, No 5-6, Aly 22, Ln 513 Ruiguang Rd., Neihu Dist.; October 7, 2015
Recombinant fusion protein comprising exenatide and XTEN Short bowel syndrome NAIA Pharmaceuticals, Inc.;215 McAllister Avenue;Kentfield, California, 94904 October 7, 2015