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
Plevitrexed Ovarian cancer BTG International Ltd;10 Fleet Place, London EC4M 7SB; May 14, 2007
Pafuramidine maleate Malaria Immtech Pharmaceuticsl, Inc.;150 Fairway Drive;Vernon Hills, Illinois, 60061 May 14, 2007
Macimorelin acetate Growth hormone deficiency Aeterna Zentaris GmbH;Weissmullerstrasse 50, D-63014 Franfurt AM Main; May 14, 2007
Peptidomimetic analog of hexarelin Diagnosis of growth hormone deficiency Aeterna Zentaris GmbH;Weissmullerstrasse 50, D-63014 Franfurt AM Main; May 14, 2007
Cyclosporine A Vernal keratoconjunctivitis Santen SAS;Batiment Genavenir IV, F-91058-Evry cedex; May 4, 2007
N-Methyl-2-[3-[((E)-2-pyridin-2-yl-vinyl)-1H-indazol-6-ylsulfanyl]benzamide Papillary thyroid cancer Pfizer, Inc.;10646 Science Center Drive;San Diego, California, 92121 May 4, 2007
Attenuated autologous cancer cells and granulocyte macrophage colony stimulating factor in combination with activated autologous blood derived T-cells Primary central nervous system malignancies TVAX Biomedical, LLC;8006 Reeder Street;Lenexa, Kansas, 66214 April 27, 2007
Sodium nitrite Cyanide poisoning Hope Pharmaceuticals;16416 N. 92nd Street;Scottsdale, Arizona, 85260 April 18, 2007
Human plasma coagulation Factor VIII and human plasma von Willebrand Factor Von Willebrand disease Octapharma USA, Inc.;121 River Street, 12th Floor;Hoboken, New Jersey, 07030 April 18, 2007
Alvocidib Prolymphocytic leukemia Tolero Pharmaceuticals, Inc.;2975 West Executive Parkway, Suite 320;Lehi, Utah, 84043 April 13, 2007
Thiotepa Conditioning treatment prior to hematopoietic stem cell transplantation ADIENNE S.A.;Via Zurigo, 46, 6900 Lugano; April 2, 2007
Sodium nitrite Sickle cell anemia Hope Pharmaceuticals;16416 N. 92nd Street;Scottsdale, Arizona, 85260 April 2, 2007
Sodium nitrite/sodium thiosulfate Cyanide poisoning Keystone Pharmaceuticals, Inc.;26072 Merit Circle;Laguna Hills, California, 92653 March 23, 2007
Rapamycin Tuberous sclerosis OncoImmune, Inc.;333 Parkland Plaza, Suite 1000;Ann Arbor, Michigan, 48103 March 20, 2007
Aminolevulinic acid hydrochloride Esophageal dysplasia DUSA Pharmaceuticals, Inc.;25 Upton Place;Wilmington, Massachusetts, 01887 March 20, 2007
Adeno-associated viral vector expressing human acid alpha glucosidase gene Pompe disease Audentes Therapeutics, Inc.;101 Montgomery St., #2650;San Francisco, California, 94104 March 20, 2007
Cisplatin in liposomal formulation Pediatric osteosarcoma Eleison Pharmaceuticals LLC;263 13th Avenue, Suite 375;St. Petersburg, Florida, 33701 March 20, 2007
Cethromycin Prophylactic treatment of patients exposed to inhalation anthrax Advanced LIfe Sciences, Inc.;1440 Davey Road;Woodridge, Illinois, 60517 February 28, 2007
Chenodeoxycholic acid Cerebrotendinous xanthomatosis Leadiant Biosciences, Inc.;9841 Washingtonian Boulevard, Suite 500;Gaithersburg, Maryland, 20878 February 12, 2007
Fenretinide Ewing's sarcoma family of tumors Cancer Research UK;61 Lincoln's Inn Fields, London WC2A 3PX; February 1, 2007
Fibrin-based agent containing a N-terminally modified parathyroid hormone fragment TGplPTH1-34 Solitary (unicameral) bone cysts Kuros Biosurgery AG;Technoparkstrasse 1;Zurich February 1, 2007
Recombinant human platelet derived growth factor BB Osteonecrosis Luitpold Pharmaceuticals, Inc.;800 Adams Avenue, Suite 100;Norristown, Pennsylvania, 19403 February 1, 2007
Brentuximab vedotin Hodgkin’s disease Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 January 30, 2007
Cytarabine liposome Gliomas Bruce Frankel, MD;Medical University of South Carolina, 96 Jonathan Lucas Street, Suite 428 CSB;Charleston, South Carolina, 29425 January 30, 2007
Sodium phenylbutyrate Spinal muscular atrophy OrphaMed, Inc.;7720 N. Dobson Road, subsidiary of Medicis Pharmaceutical Corp.;Scottsdale, Arizona, 85256 January 25, 2007