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.

Become a Partner.

Country Product Indication Sponsor Date
Adeno-associated viral serotype 2 vector under the regulatory control of a CMV promoter encoding the human tripeptidyl peptidase-1 (hTPP1) cDNA Late Infantile Neuronal Ceroid Lipofuscinosis Spark Therapeutics, Inc.;3737 Market Street, Suite 1300;Philadelphia, Pennsylvania, 19104 March 16, 2016
Trientine tetrahydrochloride Treatment of Wilson’s disease GMP-Orphan SAS;Voisin Consulting, Inc., 222 Third Street, Suite 3121;Cambridge, Massachusetts, 02142 March 10, 2016
Adenovirus associated viral vector serotype 5 containing the human RPE65 gene Leber congenital amaurosis MeiraGTx Limited;450 East 29th Street, 15th Floor;New York, New York, 10016 March 10, 2016
Adenovirus associated viral vector serotype 8 containing the human CNGB3 gene Achromatopsia MeiraGTx Limited;450 East 29th Street, 15th Floor;New York, New York, 10016 March 10, 2016
Anfibatide Thrombotic thrombocytopenic purpura Lee's Pharmaceutical (Hong Kong) Limited;Unit 110-11, Bio-Informatics Centre, Hong Kong Science Park, Shatin, Hong Kong; March 10, 2016
Rosomidnar Diffuse large B-cell lymphoma ProNAi Therapeutics, Inc.;46701 Commerce Center Drive;Plymouth, Michigan, 48170 March 10, 2016
Bevacizumab Coats Disease MicroSert, Ltd.;Bar Yehuda Street;Nesher March 10, 2016
Trientine tetrahydrochloride Treatment of Wilson�s disease GMP-Orphan SAS;Voisin Consulting, Inc., 222 Third Street, Suite 3121;Cambridge, Massachusetts, 02142 March 10, 2016
1,2:5,6-dianhydrogalactitol Medulloblastoma DelMar Pharmaceuticals, Inc.;3475 Edison Way, Suite R;Menlo Park, California, 94025 March 10, 2016
Competetive small molecule inhibitor of the enzyme O-GlcNAcase Progressive supranuclear palsy Merck & Company, Inc.;126 East Lincoln Avenue, P. O. Box 2000;Rahway, New Jersey, 07065 March 10, 2016
Selective inhibitor of fungal CYP51 Coccidioidomycosis Viamet Pharmaceuticals, Inc.;4505 Emperor Boulevard, Suite 300;Durham, North Carolina, 27703 March 3, 2016
Encochleated amikacin Non-tuberculous mycobacteria (NTM) infections Aquarius Biotechnologies, Inc.;1545 U.S. 206;Bedminster Township, New Jersey, 07921 March 3, 2016
Tesevatinib Autosomal recessive polycystic kidney disease Kadmon Corporation, LLC;450 East 29th Street;New York, New York, 10016 March 1, 2016
Bacillus subtilis oxalate decarboxylase Pediatric hyperoxaluria Allena Pharmaceuticals, Inc.;1 Executive Park Drive, Suite 202;Newton, Massachusetts, 02462 February 29, 2016
Human monoclonal antibody targeting human sclerostin Osteogenesis imperfecta Mereo Biopharma 3 Limited;1 Cavendish Place, 4th Floor;London February 29, 2016
Adeno-associated virus vector encoding the SQ variant of human blood clotting factor VIII Hemophilia A BioMarin Pharmaceutical, Inc.;105 Digital Drive;Novato, California, 94949 February 29, 2016
Sodium alginate oligosaccharide Cystic fibrosis AlgiPharma AS;Industriveien 33, N-1337;Sandvika February 24, 2016
Ombitasvir/Paritaprevir/Ritonavir Pediatric patients with chronic hepatitis C virus infection AbbVie Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 February 24, 2016
Rapamycin Tuberous sclerosis DSLP;129 Hurstmere Road, Level 1, Nielsen Building;Auckland February 24, 2016
Riluzole Spinocereballar ataxia Biohaven Pharmaceutical Holding Company, Ltd.;234 Church Street, Suite 304;New Haven, Connecticut, 06511 February 23, 2016
Growth hormone releasing hormone, 1-44 human amide Growth hormone deficiency Hollenbeck Pharmaceuticals, Inc.;P. O. Box 429;Falls Village, Connecticut, 06031 February 23, 2016
Human wilms tumor protein 1 peptides Malignant pleural mesothelioma SELLAS Life Sciences Group;75 Baarerstrasse;Zug February 23, 2016
Riluzole Spinocereballar ataxia Biohaven Pharmaceutical Holding Company, Ltd.;234 Church Street, Suite 304;New Haven, Connecticut, 06511 February 23, 2016
Cannabidiol Fragile X syndrome Zynerba Pharmaceuticals, Inc.;80 West Lancaster Avenue, Suite 300;Devon, Pennsylvania, 19333 February 23, 2016
CaspaCIDe System (combination product) consisting of allogeneic T Cell genetically modified to contain an inducible caspase 9 gene and Rimiducid Graft Versus Host Disease Bellicum Pharmaceuticals, Inc.;2130 West Holcombe Boulevard;Houston, Texas, 77030 February 18, 2016