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
Cysteamine Neuronal ceroid lipofuscinoses (Batten disease) Raptor Pharmaceuticals, Inc.;9 Commercial Blvd., Suite 200;Novato, California, 94949 August 6, 2008
Intravenous immune globulin (human) 10% Stiff-person syndrome Octapharma USA, Inc.;121 River Street;Hoboken, New Jersey, 07030 July 31, 2008
Deferiprone Friedreich Ataxia ApoPharma, Inc.;200 Barmac Drive, M9L 2Z7; July 31, 2008
4-[1]benzofuro[3,2-d]pyrimidin-4-yl-N-(1,3-benzodioxol-5-ylmethyl)piperazine-1-carbothioamide Glioblastoma SuperGen, Inc.;4140 Dublin Blvd;Dublin, California, 94568 July 30, 2008
Anthrax immune globulin (human) Toxemia associated with inhalational anthrax Cangene Corp. - Emergent Biosolutions;155 Innovation Drive, R3T 5Y3; July 29, 2008
Recombinant disintegrin and metalloprotease with thrombospondin type 1 motifs Thrombotic thrombocytopenic purpura including its congential, acquired idiopathic, and secondary forms Baxalta US Inc.;1 Baxter Way;Westlake Village, California, 91362 July 29, 2008
Anthrax immune globulin (human) Anthrax Cangene Corp. - Emergent Biosolutions;155 Innovation Drive, R3T 5Y3;Winnipeg, MB July 29, 2008
Recombinant disintegrin and metalloprotease with thrombospondin type 1 motifs Idiopathic thrombocytopenic purpura Baxalta US Inc.;1 Baxter Way;Westlake Village, California, 91362 July 29, 2008
3-fluoro-5-[5-(2-menthyl-thiazol-4-ylethylnyl)-pyridin-2-yl]-benzonitrile dihydrochloride Behavioral abnormalities associated with fragile X syndrome Seaside Therapeutics;840 Memorial Drive;Cambridge, Massachusetts, 02139 July 28, 2008
Afamelanotide Erythropoietic porphyrias Clinuvel Inc.;40 Worth Street, Suite 1053;New York, New York, 10013 July 17, 2008
[Nle4, D-Phe7]-a-melanocyte stimulating hormone Erythropoietic porphyrias Clinuvel Inc.;40 Worth Street, Suite 1053;New York, NY, 10013 July 17, 2008
Mx-dnG1 Soft tissue sarcoma Epeius Biotechnologies Corporation;475 Huntington Drive;San Marino, California, 91108 June 24, 2008
Mx-dnG1 Pediatric osteosarcoma Epeius Biotechnologies Corporation;475 Huntington Drive;San Marino, California, 91108 June 24, 2008
Carbon monoxide For use in the reduction of the incidence and severity of delayed graft function in patients undergoing solid organ transplanation INO Therapeutics;Perryville III Corporate Park, P. O. Box 9001;Hampton, New Jersey, 08827 June 24, 2008
Cicletanine hydrochloride Treatment for pulmonary arterial hypertension Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 June 24, 2008
Cardiotrophin-1 To protect the liver from ischemia-reperfusion injury inherent to the procedure of transplantation Digna Biotech, S.L.;Avenids Carlos III de Noble, Pamplona (Navarra); June 24, 2008
Alicaforsen Pouchitis Atlantic Healthcare Limited;Maple House, Halsted, Essex CO9 4BB; June 24, 2008
Recombinant adeno-associated virus retinal pigment epithelium gene vector AAV2-hRPE65v2 Leber congenital amaurosis Spark Therapeutics, Inc.;3737 Market Street, Suite 1300;Philadelphia, Pennsylvania, 19104 June 24, 2008
4-[[[4-(4-Chlorophenoxy)phenyl]sulfonyl]-methyl] tetrahydro-N-hydroxy-2H-pyran-4-carboxamide Prophylaxis of organ rejection in patients receiving allogeneic liver transplants Conatus Pharmaceuticals, Inc.;4365 Executive Drive;San Diego, California, 92121 June 18, 2008
Elacytarabine Acute myeloid leukemia Clavis Pharma ASA;Parkveien 53 B; June 18, 2008
Cenersen Metastatic Melanoma Eleos, Inc.;1001 Twelve Oaks Center Drive, Suite 1002;Wayzata, Minnesota, 55391 June 18, 2008
Azacitidine Acute myeloid leukemia Celgene Corporation;9900 W. 109th Street, Suite 300;Overland Park, Kansas, 66210 June 18, 2008
Bupivacaine Relief of persistent pain associated with postherpetic neuralgia DURECT Corporation;10260 Bubb Road;Cupertino, California, 95014 June 18, 2008
2 dimethylbutyrate Beta-thalassemia HemaQuest Pharmaceuticals, Inc.;1001 Fourth Avenue, Suite 3317;Seattle, Washington, 98154 June 18, 2008
Blinatumomab Acute lymphoblastic leukemia Amgen, Inc.;One Amgen Center Dr., Mail Stop 17-2-A;Thousand Oaks, California, 91320 May 16, 2008