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Search results for "ORPHAN DRUG" in comments (approximate match)
Status:
Possibly Marketed Outside US
Source:
Tylan 200
Source URL:
First approved in 2009
Source:
Tylosin Phosphate by Biovet Joint Stock Company
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Conditions:
Tylosin (trade names Tylocine, Tylan) is a bacteriostat feed additive used in veterinary medicine. It has a broad spectrum of activity against Gram-positive organisms and a limited range of Gram-negative organisms. It is found naturally as a fermentation product of Streptomyces fradiae. It is a macrolide antibiotic. Tylosin is used in veterinary medicine to treat bacterial infections in a wide range of species and has a high margin of safety. Tylosin is certified by the FDA but is only approved for use in livestock such as cattle, chickens, swine, and turkeys. The FDA has prohibited the use of tylosin in dogs and cats, except where it is specifically prescribed by a veterinarian. Tylosin has a bacteriostatic effect on susceptible organisms, caused by inhibition of protein synthesis through binding to the 50S subunit of the bacterial ribosome.
Status:
Possibly Marketed Outside US
Source:
NCT01904396: Phase 4 Interventional Unknown status Carnitine Deficiency
(2013)
Source URL:
First approved in 2009
Source:
IQQU Acne Serum by IQQU USA Co.
Source URL:
Class (Stereo):
CHEMICAL (RACEMIC)
Conditions:
L-carnitine (L-beta-hydroxy-gamma-N,N,N-trimethylaminobutyric acid) is conditionally necessary for mitochondrial transport and metabolism of long-chain fatty acids, and thus for myocardial energetic metabolism. D-carnitine is not biologically active and might interfere with proper utilization of the L isomer, and so there are claims that the racemic mixture (DL-carnitine) should be avoided. The pharmacological effects of carnitine are stereospecific: L-carnitine was effective in various animal and clinical studies, while D- and DL-carnitine was found to be ineffective or even toxic to some cells and tissues, such as muscle cells and the myocardium. DL-carnitine caused symptoms of myasthenia and cardiac arrhythmias, which disappeared after L-carnitine administration.
Status:
Possibly Marketed Outside US
First approved in 2007
Source:
NADA141275
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Conditions:
Emodepside is a semi-synthetic product (originated by Astellas and out-licensed to Bayer for animal and human use); its precursor is synthesized by a fungus living in the leaves of Camellia japonica. It is a potent antihelminthic drug used in combination with praziquantel (as Profender®) and in combination with toltrazuril (as Procox®) for the treatment of parasitic worms in cats and dogs. Emodepside, a semi-synthetic derivative of PF1022A, belongs to a new class of anthelmintic drugs, the cyclooctadepsipeptides, and shows good efficacy against macrocyclic lactone-, levamisole- or benzimidazole-resistant nematode populations. Although putative receptors for emodepside have already been discovered, its mode of action is still not fully understood. It has being suggested that GABA(A)-receptor UNC-49 is associated with the emodepside mode of action. It has also being shown that Emodepside binds to a presynaptic latrophilin receptor in nematodes. The following presynaptic signal transduction occurs via activation of Gqalpha protein and phospholipase-Cbeta, which leads to mobilization of diacylglycerol (DAG). DAG then activates UNC-13 and synaptobrevin, two proteins which play an important role in presynaptic vesicle-functioning. This finally leads to the release of a currently unidentified transmitter. The transmitter (or modulator) exerts its effects at the postsynaptic membrane and induces a flaccid paralysis of the pharynx and the somatic musculature in nematodes.
Status:
Possibly Marketed Outside US
Source:
NCT03915535: Phase 4 Interventional Terminated Healthy Athletes
(2019)
Source URL:
First approved in 2007
Source:
DHA by Exeltis USA, Inc
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Conditions:
Doconexent (Docosahexaenoic acid, DHA) is an omega-3 fatty acid that is a primary structural component of the human brain, cerebral cortex, skin, and retina. DHA is widely used as a food supplement, and is beleived to support healthy brain development in young childred, prevent cardiovascular disease and cognitive decline during Alzheimer's disease. Most of these claims, however, were not supported by clinical trials. DHA spray is used as a tanner.
Status:
Possibly Marketed Outside US
Source:
NCT02796794: Phase 4 Interventional Unknown status Sepsis
(2015)
Source URL:
First approved in 2007
Source:
Fosteum by Primus Pharmaceuticals, Inc.
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
GENISTEIN is an isoflavonoid derived from soy products. It inhibits protein-tyrosine kinase and topoisomerase-II (DNA topoisomerases, type II) activity and is used as an antineoplastic and antitumor agent. Experimentally, it has been shown to induce G2 phase arrest in human and murine cell lines. Additionally, genistein has antihelmintic activity. It has been determined to be the active ingredient in Felmingia vestita, which is a plant traditionally used against worms. It has also been demonstrated to be effective against intestinal parasites such as the common liver fluke, pork trematode and poultry cestode. Further, genistein is a phytoestrogen which has selective estrogen receptor modulator properties. It has been investigated in clinical trials as an alternative to classical hormone therapy to help prevent cardiovascular disease in postmenopausal women. Genistein can be found in food sources such as tofu, fava beans, soybeans, kudzu, and lupin. It is also present in certain cell cultures and medicinal plants.
Status:
Possibly Marketed Outside US
First approved in 2007
Source:
21 CFR 352
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Conditions:
Bisoctrizole (Tinosorb M) is a hybrid type sun blocking agent acting as both chemical and physical sunscreen. Bisoctrizole is a hybrid UV absorber, the only organic UV filter produced and microfine organic particles (< 200 nm), like microfine zinc oxide and titanium dioxide. Where other organic UV absorbers need to be dissolved in either the oil or water phase, bisoctrizole dissolves poorly in both. It covers both UVA and UVB range. Furthermore, it does not degrade under sunlight and is not significantly absorbed into the skin. It appear highly compatible with many other common sun blocking agents. Overall, it appears to have a good safety profile, even though current safety data are insufficient and long-term human safety studies are lacking. Unlike some other organic sunscreen actives, it shows no estrogenic effects in vitro. Bisoctrizole is not approved by the U.S. Food and Drug Administration (FDA), but is approved in the EU and other parts of the world. Bisoctrizole is highly stable and also has a stabilizing effect on other UV absorbers, particularly the UVB blocker octyl methoxycinnamate (octinoxate). Bisoctrizole appears to be relatively non-toxic and rarely causes skin irritation. As with many synthetic chemicals, it is unclear whether bisoctrizole may produce low-level skin damage or systemic effects with long-term use. Considering that bisoctrizole is stable, poorly soluble and minimally absorbed by the skin, the risks appear to be low. Still, more research is on bisoctrizole safety is needed.
Status:
Possibly Marketed Outside US
Source:
21 CFR 352
(2006)
Source URL:
First approved in 2006
Source:
21 CFR 352
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
ERGO (Ergothioneine) is naturally occurring antioxidant derived from the diet and abundantly contained in golden oyster mushroom (Pleurotus cornucopiae var. citrinopileatus. It is hydrophilic and membrane impermeable, but is well absorbed from the gastrointestinal tract due to the presence of a specific carrier‐mediated transport system, carnitine/organic cation transporter OCTN1/SLC22A4, essential for the beta-oxidation of fatty acids. Numerous in vitro assays have demonstrated the antioxidant and cytoprotective capabilities of ERGO against a wide range of cellular stressors, but an antioxidant role has yet to be fully verified in vivo. Nevertheless, the accumulation, tissue distribution, and scavenging properties, all highlight the potential for EGT to function as a physiological antioxidant. Because ERGO is highly distributed into the brain after oral ingestion, OCTN1 may contribute to the alleviation of oxidative stress and promotion of neuronal differentiation via the uptake of ERGO in the brain, perhaps abating symptoms of neurological disorders.
Status:
Possibly Marketed Outside US
Source:
NCT02356107: Phase 4 Interventional Completed Major Depressive Disorder
(2015)
Source URL:
First approved in 2004
Source:
21 CFR 352
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Creatine is an amino acid that occurs in vertebrate tissues and in urine. It is synthesized mainly in the liver from three different amino acids: arginine, glycine, and methionine. In skeletal muscles, which store 95% of it, creatine generally occurs as phosphocreatine. The rest is stored in the brain, heart, and testes. Creatine functions as part of the cell's energy shuttle. It is excreted as creatinine in the urine.
Status:
Possibly Marketed Outside US
Source:
21 CFR 348
(2004)
Source URL:
First approved in 2004
Source:
21 CFR 348
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
NAG-thiazoline (NGT) and its derivatives are well-known inhibitors against most b-acetylglucosaminidases (b-GlcNAcases) except for insect and bacterial chitinolytic b-GlcNAcase. Among them, NGT was the only one specifically designed to be an analog of the oxazolinium reaction intermediate based on the substrate-assisted mechanism, an attractive starting point to develop potent inhibitors. It is a major building block for the synthesis of potential inhibitors of transglycosylases involved in bacterial cell wall biosynthesis.
Status:
Possibly Marketed Outside US
First approved in 2003
Source:
BLA125063
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
Glutathione (GSH, also called as reduced glutathione) is a tripeptide with many roles in cells. It conjugates to drugs to make them more soluble for excretion, is a cofactor for some enzymes such as, glutathione reductase, glutathione peroxidases, peroxiredoxins. Glutathione S-transferases catalyse the conjugation of GSH via a sulfhydryl group to electrophilic centers on a wide variety of substrates in order to make the compounds more water-soluble. As a part of homeopathic product, glutathione is used for temporary relief of symptoms related to Free Radical Toxicity including tingling in hands and feet, mood changes, frequent colds, poor digestion, fatigue, and constipation. In addition, for temporary relief of pain in the back from urinating, constant urging and frequent urination, kidneys sensitive to pressure, and pain from back extending down the thigh. Glutathione is an important nutrient for brain function and loss of glutathione has been implicated in Parkinson's disease. In phase II of the clinical trial was investigated whether administration of either dose of glutathione, as a nasal spray, improves PD symptoms over time in a population of individuals with Parkinson's disease (PD). In addition in phase II of clinical trial was shown, that reduced glutathione, an ingredient of RayGel™, has been helpful in decreasing some radiation therapy side effects to the skin. Reduced glutathione plays a vital role in both making DNA and cell repair. Cystic fibrosis (CF) is the most common inherited disease among the Caucasian population with considerable morbidity and reduced life expectancy. Glutathione (GSH) represents the first-line defence of the lung against oxidative stress-induced cell injury. Therapeutic approaches with inhaled GSH could improve the reduced lung antioxidant capacity in order to counterbalance the oxidant stress linked to the chronic airway inflammation and bacterial infection.