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

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Showing 1231 - 1240 of 14117 results

Status:
Designated
Source:
EU-Orphan Drug:EU/3/18/2055
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
FDA ORPHAN DRUG:515216
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
FDA ORPHAN DRUG:157102
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

LY-156735 (TIK-301) is a melatonergic agonist and serotonergic antagonist drug. In May 2004 the FDA granted orphan drug designation to TIK-301 for the treatment of circadian sleep disorders in totally blind individuals as well as in individuals with tardive dyskinesia. Theoretically, it has an antidepressant effect.
Kifunensine is an immunoactive compound originally produced by Kitasatosporia kifunense, which displays competitive inhibition against immunosuppressive factor in tumor-bearing mice. Kifunensine has also been shown to be a potent inhibitor of the purified glycoprotein processing enzyme, mannosidase I (MAN1), specifically MAN1A1, MAN1A2, MAN1B1 and MAN1C1. Kifunensine inhibits human endoplasmic reticulum α-1,2-mannosidase I and Golgi Class I mannosidases IA, IB and IC with Ki values of 130 and 23 nM, respectively. Enzymes of this class are important factors for the biosynthesis of various types of N-linked oligosaccharide structures. Inhibition of these structures by kifunensine can interfere with cell-to-cell adhesion, targeting of lysosomal hydrolases to lysosomes, and clearance of asialoglycoproteins from the serum. Kifunensine is used to suppress Endoplasmic Reticulum-Associated Degradation (ERAD) via the inhibition of endoplasmic reticulum-associated mannosidase activity. Kifunensine has shown potential for treatment of sarcoglycanopathies and lysosomal storage disorders. Orphan designation (EU/3/11/906) was granted by the European Commission to Généthon, France, for kifunensine for the treatment of beta sarcoglycanopathy, but it was withdrawn later. Kifunensine’s use as a therapeutic is currently being researched in several conditions that benefit from its ability to inhibit mannosidase I.
Status:
Designated
Source:
FDA ORPHAN DRUG:507215
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)


Echinocandin B is a naturally occurring antibiotic, isolated from Aspergillus nidulans. It acts as an inhibitor of fungal β-1,3-glucan synthase thereby disrupting the formation of the fungal cell wall. The drug itself was not developed in the clinic, but instead, it was used in the production of other echinocandin derivatives.
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)

Showing 1231 - 1240 of 14117 results