Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

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Country Product Indication Sponsor Date
Adenovirus serotype-5 (Ad5) vector that contains a modified non-oncogenic fused early 6 (E6) and early 7 (E7) gene of the human papillomavirus (HPV); (Ad5 [E1-, E2b-]-E6/E7) Squamous cell carcinoma of head and neck Etubics Corporation;410 West Harrison Street, Suite 100;Seattle, Washington, 98119 September 4, 2014
Adenoassociated virus vector (AAV) carrying a modified AAV serotype 2 backbone and coding sequence of human thymidine phosphorylase preceded by a human thyroxin-binding globulin promoter For the treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy (MNGIE) Columbia University Medical Center;Department of Neurology, 630 West 168th Street, P&S; 4-423;New York, New York, 10032 September 4, 2014
2-{[(1r,4r)-4-(((4-chlorophenyl)(phenyl)carbamoyloxy)methyl)cyclohexy]methoxy} acetic acid Pulmonary Arterial Hypertension Arena Pharmaceuticals, Inc.;6154 Nancy Ridge Drive;San Diego, California, 92121 August 28, 2014
Methotrexate Myasthenia gravis Myasthenia gravis The Universtiy of Kanasa Medical Center;3901 Rainbow Blvd, MSN 2012;Kanasa City, Kansas, 66160 August 28, 2014
Spanlecortemlocel Acute lymphoblastic leukemia Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 August 27, 2014
(3S)-1-azabicylo[2.2.2]oct-3yl {2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate Fabry disease Genzyme Corporation;500 Kendall Street;Cambridge, Massachusetts, 01242 August 26, 2014
Gefitinib Non-small cell lung cancer AstraZeneca Pharmaceuticals LP;1800 Concord Pike, P. O. Box 8355;Wilmington, Delaware, 19803 August 26, 2014
Venglustat Fabry disease Genzyme Corporation;500 Kendall Street;Cambridge, Massachusetts, 01242 August 26, 2014
Cannabidiol Glioblastoma Insys Development Company, Inc.;1333 S. Spectrum Blvd, Suite 100;Chandler, Arizona, 85286 August 20, 2014
Altiratinib Glioblastoma Deciphera Pharmaceuticals, LLC;643 Massachusetts, Suite 200;Lawrence, Kansas, 66044 August 19, 2014
Lacnotuzumab Pigmented villonodular synovitis, including giant cell tumor of the tendon sheath Novartis Pharmaceuticals Corp;One Health Plaza;East Hanover, New Jersey, 07936 August 19, 2014
Dexrazoxane hydrochloride Prevention of cardiomyopathy for chilren and adolescents 0 through 16 years of age treated with anthracyclines Satiscor, LLC;45 Marine Road;Boston, Massachusetts, 02127 August 19, 2014
Immune Globulin Subcutaneous (Human), 20% Liquid Chronic inflammatory demyelinating polyneuropathy CSL Behring;P. O. Box 61501, 1020 First Avenue;King of Prussia, Pennsylvania, 19406 August 18, 2014
Anti-Beta1 integrin monoclonal antibody Glioblastoma OncoSynergy, Inc.;409 Illinois Street, Room 3044;San Francisco, California, 94158 August 7, 2014
Dry extract from Betulae Cortex (birch bark) Dystrophic epidermolysis bullosa Birken AG;Streiflingsweg 11;Niefern-Oeschelbronn August 7, 2014
N1,N14,-diethyl-3,12,-dihydroxyhomospermine Pancreatic Cancer Sun BioPharma, Inc.;712 Vista Blvd, Suite 305;Waconia, Minnesota, 55387 August 7, 2014
Roseburia hominis Ulcerative colitis 4D Pharma Research Ltd;Life Sciences Innovation Bldg, Cornhill Road; August 4, 2014
Binimetinib Ovarian cancer Array Biopharma, Inc.;3200 Walnut Street;Boulder, Colorado, 80301 July 31, 2014
Antibody drug conjugate consisting of a humanized IgG1 anti-NaPi2b monoclonal antibody and monomethyl auristatin E Ovarian cancer Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 July 29, 2014
Antibody drug conjugate consisting of fully human anti-guanylyl cyclase C monoclonal antibody linked to the cytotoxic drug monomethyl auristatin E Stomach Cancer Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 July 21, 2014
2-[(6S)-4-(4-chlorophenyl)-2,3,9-trimethyl-6H-thienol[3,2,-f]-[1,2,4]triazolo[4,3-a][1,4]diazepin-6-yl]-N-(4-hydroxyphenyl)-acetamide dihydrate Acute myeloid leukemia OncoEthix GmbH (Merck Subsidiary);Weystrasse 20, 6000 Lucerne; July 16, 2014
Paclitaxel-coated buttress For use as local administration at the time of curative surgery in the treatment of stage 0 to III non-small cell lung cancer AcuityBio, Inc.;200 Upland Road;Newton, Massachusetts, 02460 July 15, 2014
Adalimumab Behçet’s Disease Mucora;32b Eisenberg St, Rehovot 7628810; July 10, 2014
Adeno-associated viral vector serotype 2 containing the humanREP1gene Choroideremia NightstaRx Ltd. July 4, 2014
Carbenoxolone Huntington disease Oxalys Pharmaceuticals, Inc.;4 Mollard Road;Toronto, ON M1S 2L4 July 2, 2014