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
Oral testosterone Constitutional delay of growth and puberty in adolescent boys 14 to 17 years of age TesoRx Pharma, LLC;3670 West Temple Avenue, Suite 270;Pomona, California, 91768 June 2, 2016
Human donor hematopoietic stem and progenitor cells (HSPC) that have been treated ex vivo with TBX-4000 Enhancement of cell engraftment in patients receiving hematopoietic stem cell transplant Taiga Biotechnologies, Inc.;12635 Montview Boulevard, Fitzsimmons BBioscience Park Center;Aurora, Colorado, 80045 June 2, 2016
Methimazole and liothyronine sodium Glioblastoma Musli Thyropeutics Ltd;6 HaGefen Street;zur Moshe June 2, 2016
D-methadone Postherpetic neuralgia Relmada Therapeutics, Inc.;750 Third Avenue, 9th Floor;New York, New York, 10017 June 2, 2016
Bupivacaine hydrochloride Treatment of pain associated with postherpetic neuralgia Grace Therapeutics, LLC;675 U.S. 1;North Brunswick Township, New Jersey, 08902 May 31, 2016
Rovalpituzumab tesirine Small cell lung cancer AbbVie Ltd May 30, 2016
Fc- and CDR-modified humanised monoclonal antibody against C5 Paroxysmal nocturnal hemoglobinuria Alexion Europe SAS May 30, 2016
Vemurafenib Langerhans’ cell histiocytosis Groupe d'étude des histiocytoses May 30, 2016
H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate Amyotrophic lateral sclerosis QRC Consultants Ltd May 30, 2016
Polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase Homocystinuria Alan Boyd Consultants Ltd May 30, 2016
Sodium nitrite and ethylenediaminetetraacetic acid Cystic fibrosis Arch Bio Ireland Ltd May 30, 2016
Temsirolimus X-linked adrenoleukodystrophy Consorcio Centro de Investigación Biomédica en Red M.P. May 30, 2016
4-[(2E)-1-oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide Retinitis pigmentosa Shire Pharmaceuticals Ireland Limited May 30, 2016
Arimoclomol citrate Inclusion Body Myositis Orphazyme ApS May 30, 2016
Autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene Beta thalassaemia intermedia and major GlaxoSmithKline Trading Services Limited May 30, 2016
Recombinant adeno-associated viral vector containing the humanRPGRgene Retinitis pigmentosa TMC Pharma Services Ltd May 30, 2016
Rimiducid Graft Versus Host Disease Bellicum Pharma Limited May 30, 2016
(R)-6-(2-fluorophenyl)-N-(3-(2-((2-methoxyethyl)amino)ethyl)phenyl)-5,6-dihydrobenzo[h]quinazolin-2-amine dihydrochloride Biliary tract cancer QRC Consultants Ltd May 30, 2016
Diazepam subcutaneous injection Acute repetitive seizures Xeris Pharmaceuticals, Inc.;3208 Red River Street, Suite 300;Austin, Texas, 78705 May 26, 2016
Recombinant, non-pathogenic poliovirus:rhinovirus chimera that consists of the genome of the live attenuated poliovirus serotype 1 vaccine with its cognate IRES element replaced with that of HRV2 Glioma Istari Oncology, Inc.;Duke University Medical Center, 177 MSRB, Box 3156;Durham, North Carolina, 27710 May 26, 2016
Antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA Leber's congenital amaurosis ProQR Therapeutics IV B.V.;Zernikedreef 9, 2333 CK Leiden;Leiden May 25, 2016
Non-replicating recombinant adeno-associated viral vector, serotype 8, expressing the 1A1 isoform of the bilirubin-uridine diphosphate glucuronosyltransferase gene Crigler Najjar syndrome Audentes Therapeutics, Inc.;600 California Street, 17th Floor;San Francisco, California, 94109 May 24, 2016
9-cis, 12-cis-11,11-d2-linoleic acid ethyl ester Friedreich Ataxia Retrotope, Inc.;4300 El Camino Real, Suite 201;Los Altos, California, 94022 May 23, 2016
Brentuximab vedotin Adult T-cell leukemia/lymphoma Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 May 23, 2016
Nanocomplex consisting of a small interfering RNA that targets the human EV11 oncogenic transcription factor and an elastin-like polypeptide drug carrier. Ovarian cancer PeptiMed;922 Walsh Road, Suite 208;Madison, Wisconsin, 53714 May 23, 2016