U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Showing 1911 - 1920 of 21518 results

Status:
Designated
Source:
FDA ORPHAN DRUG:670418
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
EU-Orphan Drug:EU/3/15/1481(POSITIVE)
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)

BN-82451 belongs to a family of small molecules designated as multitargeting or hybrid molecules. BN-82451 is orally active, has good central nervous system penetration, and elicits potent neuronal protection and antiinflammatory properties. BN-82451 acts via three major pathways involved in neuronal death: excito-toxicity, oxidative stress, and inflammation, and is also a mitochondrial protective agent. Because BN-82451 is a multitargeting agent, each of its specific sites of action has been extensively evaluated, namely, neuronal excitotoxicity (sodium channel blocker), oxida-tive stress (antioxidant), neuroinflammation (cyclooxygenase inhibitor), and mitochondrialdysfunction (mitochondria-protective properties). BN-82451 was found to exert a significant protection in experimental animal models mimicking aspects of cerebral ischemia, Parkinson disease, Huntington disease, and more particularly amyotrophic lateral sclerosis. BN-82451 is in phase II clinical trials by Ipsen Pharma for the treatment of Huntington’s disease. In 2015, orphan drug designation was assigned in the U.S. to the compound for the indication.
Status:
Designated
Source:
FDA ORPHAN DRUG:18887
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

B-Trisaccharide belongs to the class of organic compounds known as fatty acyl glycosides of mono- and disaccharides. B-Trisaccharide in blood group B is excreted when an individual is given Galactose. The B-trisaccharide was the only oligosaccharide detected in plasma after galactose administration to a blood-group-B secretor individual. The structure of the branched blood group B trisaccharide alone has been studied both by X-ray crystallography and by NMR.
Status:
Designated
Source:
FDA ORPHAN DRUG:211705
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)


Solamargine is the one of major compounds of Solanum lycocarpum- fruit glycoalkaloid extract, and a major steroidal alkaloid glycoside, which is purified from Solanum nigrum L (SNL), a traditional Chinese medicinal herb. It has been shown that solamargine has anti-tumor activity against the several types of cancers. Also as a part of SR-T100 Gel solamargine in the phase II of clinical trial for the treatment of Actinic Keratosis and Bowen's Disease. The precise mechanism of its actions is still undefined, but existed several potential pathways. In case of castration-resistant prostate cancer cells, solamargine inhibits the growth cells through AMPKalpha-mediated inhibition of p65, followed by reduction of MUC1 expression in vitro and in vivo. In case of lung cancer cells solamargine inhibits the growth cells through reduction of EP4 protein expression, followed by increasing ERK1/2 phosphorylation. The inter-correlations between EP4, DNA methyltransferase 1 (DNMT1) and c-Jun and feedback regulation of ERK1/2 by c-Jun contribute to the overall responses of solamargine in this process.
Status:
Designated
Source:
FDA ORPHAN DRUG:761620
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
FDA ORPHAN DRUG:18086
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

A-TRISACCHARIDE (or Blood group A trisaccharide), a core antigen fragment in ABO blood group system. It was found to be a major urinary carbohydrate depending on diet and blood type.
Status:
Designated
Source:
FDA ORPHAN DRUG:452214
Source URL:

Class (Stereo):
CHEMICAL (MIXED)

Status:
Designated
Source:
FDA ORPHAN DRUG:240207
Source URL:

Class (Stereo):
CHEMICAL (RACEMIC)

Plevitrexed is an orally bioavailable, small molecule, non-polyglutamatable, antifolate quinazoline derivative thymidine synthetase inhibitor with potential antineoplastic activity. This compound belongs to the class of organic compounds known as hippuric acids, which consist of a benzoyl group linked to the N-terminal of a glycine. Plevitrexed is transported into the cell via the physiological reduced folate carrier (RFC) system. Intracellularly, this agent selectively binds to the folate binding site of thymidylate synthase and inhibits thymidine synthesis, which may result in DNA synthesis inhibition and apoptosis. Plevitrexed has been investigated for use/treatment in pancreatic cancer, solid tumors, gastric cancer, lung cancer, and colorectal cancer.
Status:
Designated
Source:
FDA ORPHAN DRUG:312010
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Epitalon (amino acid sequence Ala-Glu-Asp-Gly) is a synthetic peptide that has been suggested to induce the activation of ribosomal genes, decondensation of pericentromeric structural heterochromatin and the release of genes repressed due to the age-related condensation of euchromatic chromosome regions. The researchers who generated Epitalon also reported an inhibitory effect on the development of spontaneous tumors in mice, and claimed that it increases neuronal activity and can be used in the treatment of Retinitis Pigmentosa. The peptide is still under pre‐clinical and clinical development and thus has not been approved for any therapeutic and/or prophylactic use by any government health authority in the USA or Europe.
Status:
Designated
Source:
FDA ORPHAN DRUG:625017
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Showing 1911 - 1920 of 21518 results