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Restrict the search for
beta carotene
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Status:
Designated
Source:
FDA ORPHAN DRUG:452214
Source URL:
Class (Stereo):
CHEMICAL (MIXED)
Status:
Designated
Source:
EU-Orphan Drug:EU/3/10/816(POSITIVE)
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
7β-hydroxycholesteryl-3-oleate has been shown to inhibit astrogliosis and intracranial glioblastoma growth. Local administration of 7β-hydroxycholesteryl-3-oleate inhibits growth of experimental rat C6 glioblastoma. These data suggest that 7β-hydroxycholesteryl-3-oleate might be useful for local glioblastoma chemotherapy. 7β-hydroxycholesteryl-3-oleate is a potent inhibitor of the endogenous cholesterol biosynthesis in brain showing a correlation between cholesterogenesis and reactive astrocyte proliferation. 7β-hydroxycholesteryl-3-oleate can reduce the astrocytic reaction following spinal cord injury, promoting the serotonergic reinnervation of a denervated territory. On 17 December 2010, orphan designation (EU/3/10/816) was granted by the European Commission to Intsel Chimos SA, France, for 7-beta-hydroxy cholesteryl-3-beta-oleate for the treatment of glioma. The substance is going to be injected directly into the brain tumour contained within liposomes, which are expected to carry the medicine into the glioma cells.
Status:
Designated
Source:
FDA ORPHAN DRUG:344011
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Ribavirin elaidate (CP-4033; TRX-201), a Lipid Vector Technology derivative of ribavirin. It inhibits elongation factor F4E (elF4E), which stimulates cell growth. Translational Therapeutics Inc. received orphan drug status from the U.S. Food and Drug Administration (FDA) in 2011 for ribavirin elaidate in the treatment of aggressive follicular, medullary and anaplastic thyroid carcinoma.
Status:
Designated
Source:
FDA ORPHAN DRUG:625017
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Status:
Designated
Source:
FDA ORPHAN DRUG:321010
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
GUANOSINE 5'-DIPHOSPHO-β-L-FUCOSE is classified as a member of the Purine nucleotide sugars. Guanosine 5'-diphospho-β-L-fucose sodium salt sodium salt is a substrate for fucosyltransferase.
Status:
Designated
Source:
FDA ORPHAN DRUG:390013
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Bromopyruvate is an halogenated analogue of pyruvic acid known as an alkylating agent reacting with thiol groups of many proteins. Bromopyruvate exerts anticancer action. It is based on the impairment of energy metabolism of tumor cells by inhibiting enzymes in the glycolysis pathway (hexokinase II, glyceraldehyde 3-phosphate dehydrogenase, phosphoglycerate kinase) and the oxidative phosphorylation (succinate dehydrogenase). Bromopyruvate induces endoplasmic reticulum stress, inhibits global protein synthesis further contributing to cancer cell death. Treatment with bromopyruvate has been administered in several cancer type models both in vitro and in vivo, either alone or in combination with other anticancer therapeutic approaches. These studies clearly demonstrate bromopyruvate broad action against multiple cancer types. This compound has also antifungal and antiparasitic activity.
Status:
Designated
Source:
FDA ORPHAN DRUG:133100
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Conditions:
3,3',5,5'-Tetraiodothyroacetic acid (Tetrac) is a deaminated analog of L-thyroxine (T4) that blocks the proangiogenesis actions of T4 and 3, 5, 3’-triiodo-L-thyronine as well as other growth factors at the cell surface receptor for thyroid hormone on integrin αvβ3. 3,3',5,5'-Tetraiodothyroacetic acid blocks the transcriptional activities directed by L-thyroxine (T4) and 3,5,3’-triiodo-L-thyronine (T3) at αvβ3, but, independently of T4 and T3, 3,3',5,5'-Tetraiodothyroacetic acid modulates transcription of cancer cell genes that are important to cell survival pathways, control of the cell cycle, angiogenesis (VEGFA, FGF), apoptosis, cell export of chemotherapeutic agents, and repair of double-strand DNA breaks. 3,3',5,5'-Tetraiodothyroacetic acid was found to perturb the angiogenesis process stimulated by VEGF (Vascular Endothelial Growth Factor) or FGF (Fibroblast Growth Factor) without influencing the preexisting blood vessels.
Status:
Designated
Source:
FDA ORPHAN DRUG:67292
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
5,6-dihydro-5-azacytidine (DHAC) is a hydrolytically stable congener of 5-azacytidine. It exerts cytostatic action in vitro. DHAC induces DNA hypomethylation. DHAC inhibits DNA methyltransferase, thereby interfering with abnormal DNA methylation patterns that are associated with genetic instability in some tumor cells. Inhibition of this enzyme may restore expression of tumor-suppressor genes and result in antitumor activity. It was in phase II studies for the treatment of several cancers such as malignant melanoma, mesothelioma and others.
Status:
Designated
Source:
EU-Orphan Drug:EU/3/16/1618
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Status:
Designated
Source:
FDA ORPHAN DRUG:71792
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Isobutyramide (VX-366) is an orally available branched chain amide that may offer an alternative to current treatments for beta-hemoglobinopathy. Isobutyramide has been shown to increase fetal hemoglobin (HbF) in patients with beta-hemoglobinopathies. Isobutyramide was originated at the Children's Hospital Oakland Research Institute which, in 1993, granted exclusive worldwide rights for the agent to Vertex. However, development of Isobutyramide has been discontinued for sickle cell anaemia and thalassaemia.
