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.

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Product Indication Date Designated
cytarabine-beta-L-aspartic acid Acute Myeloid Leukemia May 14, 2019
cereblon enzyme 3 (E3) ubiquitin ligase modulating agent Multiple myeloma May 14, 2019
genetically engineered herpes simplex virus Medulloblastoma May 09, 2019
iloprost Systemic sclerosis May 09, 2019
Human cytomegalovirus (HCMV) internal repeat short 1 (IRS1)-expressing oncolytic Herpes Simplex Virus Malignant glioma May 09, 2019
faceted gold nanocrystals Amyotrophic Lateral Sclerosis May 08, 2019
Autologous T cells transfected with PiggyBac transposon plasmid vector expressing B-cell maturation antigen (CAR-T cells) Multiple Myeloma May 08, 2019
A potent and selective inhibitor of Gamma Secretase-mediated NOTCH signaling Adenoid cystic carcinoma May 08, 2019
(6-(4-((4-(1H-pyrazol-4-yl)phenyl)amino)pyrimidin-2-yl)-1-methyl-1H-indol-2-yl)(3,3-difluoroazetidin-1-yl)methanone Idiopathic Pulmonary Fibrosis May 08, 2019
Recombinant fully human IgG1 monoclonal antibody targeted to the Zaire ebolavirus glycoprotein Ebola Virus Disease May 08, 2019
human glucagon-like peptide-2 analogue linked to a human immunoglobulin Fc fragment (ready to use pre-filled syringe) Short bowel syndrome May 01, 2019
Purified Allogeneic Human Pancreatic Islets of Langerhans Brittle Type 1 Diabetes May 01, 2019
small molecule inhibitor of KRASG12C (Kirsten rat sarcoma viral oncogene homolog) mutant protein KRAS p.G12C-positive non-small cell lung cancer May 01, 2019
allogeniec Human Pancreatic Islets of Langerhans Brittle type 1 diabetes May 01, 2019
allogeneic skin-derived ABCB5-positive mesenchymal stem cells Epidermolysis Bullosa May 01, 2019
N-{2-[N-((3S)(3-piperidyl))carbamoyl]-5-[2-(3-fluorophenyl)ethynyl](3-thienyl)}aminoamide Acute myeloid leukemia Apr 29, 2019
recombinant adeno-associated viral vector with human methylmalonyl-COA mutase (MUT) gene Methylmalonic acidemia Apr 22, 2019
vardenafil Pulmonary Arterial Hypertension Apr 22, 2019
Dexpramipexole Hypereosinophilic syndrome (HES) Apr 22, 2019
Autologous enriched T-cells genetically modified with a retroviral vector to express two chimeric antigen receptors (CARs) targeting cluster of differentiation (CD) 19 and CD 22. Acute Lymphoblastic Leukemia Apr 17, 2019