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
Bendamustine for 50ml admixture Follicular lymphoma Eagle Pharmaceuticals, Inc.;50 Tice Blvd, Suite 315;Woodcliff Lake, New Jersey, 07677 July 2, 2014
Bendamustine for 50 ml admixture Chronic lymphocytic leukemia Eagle Pharmaceuticals, INc.;50 Tice Blvd, Suite 315;Woodcliff Lake, New Jersey, 07677 July 2, 2014
Cannabidiol Dravet Syndrome Insys Development Company, Inc.;1333 S. Spectrum Blvd, Suite 100;Chandler, Arizona, 85286 July 1, 2014
2-(2-phenylvinyl)-4-[4-methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt Hepatocellular Carcinoma CASI Pharmaceuticals, Inc.;9620 Medical Center Drive, Suite 300;Rockville, Maryland, 20850 July 1, 2014
Lumacaftor/ivacaftor Cystic fibrosis Vertex Pharmaceuticals Inc.;50 Northern Avenue;Boston, Massachusetts, 02210 June 30, 2014
Clofazimine Active tuberculosis Novartis Pharmaceuticals Corp.;One Health Plaza, Bldg 135/409;East Hanover, New Jersey, 07936 June 25, 2014
Cannabidiol Lennox-Gastaut syndrome Insys Development Company, Inc.;1333 S. Spectrum Blvd, Suite 100;Chandler, Arizona, 85286 June 23, 2014
(2’R,3’S)-2′-hydroxy-N-carboxy-3′-amino-5′-methyl-hexanoic,N-tert-butyl ester, 13 ester 5B-20-epoxy-1B,2a,4a,7B,9a,10a,13a-heptahydroxy-4,10-diacetate-2-benzoate-(1″S)-7,9-acrolein acetal-11(15-1)-abeotaxane Progressive supranuclear palsy Cortice Biosciences, Inc.;1345 Avenue of the Americas, 42nd Floor;New York, New York, 10105 June 23, 2014
Re188 P2045 somatostatin peptide analogue Pancreatic cancer Andarix Pharmaceuticals, Inc.;141 Powderhouse Blvd, Suite 1;Somerville, Massachusetts, 02144 June 19, 2014
Carglumic acid Organic acidemias Orphan Europe SARL;Immeuble Le Wilson, 70 avenue du General de Gaulle;92800 Puteaux June 17, 2014
6-[4-Deoxy-4-[(2E,4E)-tetradecadienoylglycyl]amino-L-glycero-B-L-manno-heptopyranosyl]amino-9H-purine Multiple myeloma DARA Therapeutics, Inc.;8601 Six Forks Road, Suite 160 (Forum 1);Raleigh, North Carolina, 27615 June 12, 2014
Epstein-Barr virus-specific autologous cytotoxic T lymphocytes Epstein-Barr virus positive head and neck cancers (which includes nasopharyngeal carcinoma) Tessa Therapeutics Pte.;8 Temasek Blvd #24-02, Suntec Tower Three; June 12, 2014
Mocetinostat Myelodysplastic syndrome Mirati Therapeutics, Inc.;9363 Towne Centre Drive, Suite 200;San Diego, California, 92121 June 12, 2014
Enasidenib Acute myeloid leukemia Celgene Corporation;Corporate Woods Bldg 32, Suite 900, 9225 Indian Creek Parkway;Overland Park, Kansas, 66210 June 12, 2014
Eculizumab Myasthenia gravis Myasthenia gravis Alexion Pharmaceuticals, Inc.;100 College Steet;New Haven, Connecticut, 06510 June 12, 2014
Autologous genetically modified human dermal fibroblasts Dystrophic epidermolysis bullosa Fibrocell Technologies, Inc.;405 Eagleview Blvd;Exton, Pennsylvania, 19341 June 10, 2014
(5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-ß-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9-tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one Biliary tract cancer Mundipharma Corporation Limited June 4, 2014
Afatinib Malignant brain and central nervous system tumors Boehringer Ingelheim Pharmaceuticals, Inc.;900 Ridgebury Road, P. O. Box 368;Ridgefield, Connecticut, 06877 June 4, 2014
Adeno-associated viral vector serotype 2 containing the humanCHMgene encoding human Rab escort protein 1 Choroideremia Alan Boyd Consultants Ltd June 4, 2014
(5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-beta-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9-tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one Biliary tract cancer Mundipharma Corporation Limited June 4, 2014
Adeno-associated viral vector serotype 2 containing the humanCHMgene encoding human Rab escort protein 1 Choroideremia Spark Therapeutics Ireland Ltd June 4, 2014
Everolimus Diffuse large B-cell lymphoma Novartis Pharmaceuticals Corp.;One Health Plaza;East Hanover, NJ, 07936-1080 June 2, 2014
Echothiophate iodide Stargardt's disease Makindus, Inc.;3805 Old Easton Road;Doylestown, Pennsylvania, 18902 June 2, 2014
Everolimus Diffuse Large B-cell Lymphoma Novartis Pharmaceuticals Corp.;One Health Plaza;East Hanover, New Jersey, 07936 June 2, 2014
2-[4-(1-Methyl-4-pyridin-4-yl-lH-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid Huntington disease Pfizer Inc.;445 Eastern Point Road;Groton, Connecticut, 06340 June 2, 2014