Close
Close
Treatment Database

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.

Product Indication Date Designated
1-(((Z)-hexadec-1-en-1-yl)oxy)-3-((2-oxido-1,3,2-oxazaphospholidin-2-yl)oxy)propan-2-yl (4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenoate Rhizomelic Chondrodysplasia Punctata Aug 08, 2019
Lentiviral vector expressing human fumarylacetoacetate hydrolase Hereditary tyrosinemia type I Aug 08, 2019
4-(4-((4-chloro-3-(trifluoromethyl)phenyl)sulfonyl)phenyl)-2,4-dihydro-3H-1,2,4-triazole-3-thione Amyotrophic lateral sclerosis Aug 08, 2019
(R)-tetrahydrofuran-3-yl 4-(6-(5-(4-ethoxy-1-isopropylpiperidin-4-yl)pyridin-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate Fibrodysplasia ossificans progressiva Aug 08, 2019
(2R,3S)-2-(3-(4,5-dichloro-1H-benzo[d]imidazol-1-yl)propyl)piperidin-3-ol Hydrogen Chloride Idiopathic pulmonary fibrosis Aug 05, 2019
antisense oligonucleotide (5� AsGsTsTsGsGsAsGsCsAsAsGsAsTsTsAsTsC 3�) Dravet syndrome Aug 05, 2019
Temozolomide Neuroblastoma Aug 01, 2019
1-{4-[2-(5-ethoxymethyl-2-methyl-phenylamino)-oxazol-5-yl]-phenyl}-imidazolidin-2-one Acute myeloid leukemia Aug 01, 2019
heparin sodium Cystic Fibrosis Jul 29, 2019
elafibranor Primary Biliary Cholangitis Jul 25, 2019
(S)-4,11-diethyl-4-hydroxy-3,14-dioxo-3,4,12,14-tetrahydro-1H-pyrano[3',4':6,7]indolizino[1,2-b]quinolin-9-yl 4-(2-(5-(3-(2,4-dihydroxy-5-isopropylphenyl)-5-hydroxy-4H-1,2,4-triazol-4-yl)-1H-indol-1-yl)ethyl)piperidine-1-carboxylate Pancreatic cancer Jul 24, 2019
(R)-6-((3,3-difluoro-1-methylpiperidine-4-yl)oxy-N-(3-ethynyl-2-fluorophenyl)-7-methoxyquinazolin-4-amine-,fumerate (2:1) (salt) Glioma (including glioblastoma and anaplastic astrocytoma) Jul 24, 2019
Episcleral Topotecan Retinoblastoma Jul 24, 2019
aflibercept Retinopathy of Prematurity Jul 23, 2019
recombinant human anti-human sortilin (SORT1) IgG1 G1m17,1 [or G1m (z,a)] kappa monoclonal antibody Frontotemporal Dementia Jul 22, 2019
pozelimab Paroxysmal Nocturnal Hemoglobinuria Jul 22, 2019
N-(4-amino-3,4-dioxo-1-phenylbutan-2-yl)-4-(2-fluorophenyl)-2-methyloxazole-5-carboxamide Idiopathic Pulmonary Fibrosis Jul 22, 2019
concizumab Hemophilia B Jul 22, 2019
autologous T-cells genetically modified to express a CD123-specific, hinge-optimized, CD28-costimulatory chimeric receptor and a truncated human epidermal growth factor receptor Acute myeloid leukemia Jul 22, 2019
artesunate Malaria Jul 22, 2019