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

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Showing 581 - 590 of 9908 results

Status:
Designated
Source:
FDA ORPHAN DRUG:292809
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

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:449014
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

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

Class (Stereo):
CHEMICAL (ABSOLUTE)

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: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:890422
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)



GGTI-2418, also known as PTX100, is a synthetic peptidomimetic inhibitor of geranylgeranyltransferase I (GGTase I) that appears to induce apoptosis by downregulating several pivotal oncogenic and tumor survival pathways. In preclinical studies, GGTI-2418 has been shown to cause significant breast tumor regression in ErbB2 transgenic mice model. GGTI-2418 was the first GGTase I inhibitor to enter clinical development in early 2009. Phase I clinical trials early results demonstrated that ~30% of patients with advanced solid tumors had stable disease following GGTI-2418 therapy, the compound was well-tolerated and had minimal toxicity. However, the Phase I trial of GGTI-2418 has been stopped due to its lack of efficacy in patients. In February 2015 Prescient Therapeutics in-licensed the p27 biomarker for use as a companion diagnostic. Patients with low levels of p27 are more likely to respond to GGTI-2418 therapy.
Status:
Designated
Source:
EU-Orphan Drug:EU/3/06/386
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
FDA ORPHAN DRUG:214405
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

L-aminocarnityl-succinyl-leucyl-argininal-diethylacetal (Myodur or C101) is a compound that comprise a carrier residue that is an analog of carnitine, a linker group, and a residue of a protease inhibitor comprising an acetal derivative of the aldehyde group. Myodur is a muscle cell targeted product that inhibits calpain in an effort to preserve muscle tissue. In 2006 CepTor Corporation (OTCBB:CEPO), a development-stage biopharmaceutical company focusing on cell-targeted therapeutic products for neuromuscular and neurodegenerative diseases announced that the Office of Orphan Products Development of the Food and Drug Administration (FDA) granted orphan drug designation for Myodur for both Duchenne and Becker muscular dystrophies. Myodur development has been discontinued.