- Home
- /
- Treatment
- /
- Page 39
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 |