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Restrict the search for
m nalidixic acid
to a specific field?
Status:
Designated
Source:
FDA ORPHAN DRUG:890422
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
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:
FDA ORPHAN DRUG:269808
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
TH9402 is a dibrominated rhodamine derivative and potent photosensitizer and useful in photodynamic therapy. TH9402 preferentially localizes in mitochondria and when exposed to 500- to 600-nm wavelength visible light delivered through the Theralux device, it becomes highly cytotoxic through oxidative damage. TH9402 eradicates multiple myeloma (MM) and BC cell lines, while sparing more than 50% of normal pluripotential blood stem cells from healthy volunteers. TH9402 offers an excellent potential as an ex vivo photodynamic purging agent for autologous transplantation in multiple myeloma and breast cancer treatment. TH9402 photodynamic-cell-therapy process conditions recognized as safe for normal human haematopoietic stem cells and progenitors demonstrated the efficacy of the purging procedure on various leukaemias.
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: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:517616
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Florilglutamic acid enters cells through a transport system known as xC- (cystine/glutamate antiporter) which is more abundant in cancer tissue. Florilglutamic acid (18F) is a radioactive compound for use with an imaging method known as positron
emission tomography (PET). When injected into the patient, florilglutamic acid (18F) is
more effectively taken up by the cancer cells in the liver from where it is expected to emit radiation
that can be detected in a PET scan, thereby allowing the cancer to be diagnosed. Orphan designation (EU/3/16/1632) was granted by the European Commission to Piramal Imaging GmbH, Germany, for florilglutamic acid (18F) for the diagnosis of hepatocellular carcinoma.
Status:
Designated
Source:
FDA ORPHAN DRUG:434014
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
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:
EU-Orphan Drug:EU/3/15/1449
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
Myriocin ((2S,3R,4R,6E)-2-amino-3,4- dihydroxy-2-(hydroxymethyl)-14-oxo-6-eicosenoic acid, ISP-1, thermozymocidin) is a small-molecule immunosuppressant, isolated from the Mycelia sterilia thermophilic fungus. Myriocin inhibits serine palmitoyltransferase (SPT) at picomolar concentrations blocking synthesis of ceramide, a precursor of sphingomyelin (SM) and glycosphingolipids. Inhibition of hepatic serine palmitoyl transferase reduces plasma sphingomyelin levels in the absence of changes in cholesterol or triglyceride (TG) concentration and this leads to a reduction of atherosclerosis. In preclinical studies, Myriocin treated mice shows significant reductions in both plasma SM and Glycosphingolipids (GSL) concentration. Moreover, SM and GSL concentrations were significantly correlated, indicating that SPT inhibition suppresses the synthesis of both these sphingolipids concomitantly. The inhibition of atherosclerosis induced by myriocin was not associated with changes in plasma cholesterol or TG concentrations or lipoprotein profiles.
Status:
Designated
Source:
FDA ORPHAN DRUG:705019
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
