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Search results for "ORPHAN DRUG|Designated|Treatment of Prader-Willi Syndrome" in comments (approximate match)
Status:
Designated
Source:
FDA ORPHAN DRUG:95896
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Conditions:
Etiocholanedione is a natural metabolite of dehydroepiandrosterone with potent antiobesity activity. Etiocholanedione has been identified as a metabolite of an altered androgen metabolism that eventually leads hepatocellular carcinoma to impaired hormone responsiveness in human. Etiocholanedione has been identified as a metabolite of 17alpha-hydroxyprogesterone in some patients affected by congenital adrenal hyperplasia, although it doesn't appear to account for the masculinization observed in congenital hyperplasia.
Status:
Possibly Marketed Outside US
Source:
NCT04482959: Phase 4 Interventional Completed Myoma;Uterus
(2020)
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Conditions:
Carbetocin is a synthetic analogue of human peptide hormone, oxytocin. Like oxytocin, it stimulate oxytocin receptors and is used to facilitate childbirth. The drug is being marketed in Europe under the name Pabal for the prevention of uterine atony following delivery of the infant by Caesarean section. If untreated by carbetocin, uterine atony may lead to postpartum haemorrhage.
Status:
Possibly Marketed Outside US
Source:
NCT04614233: Phase 4 Interventional Terminated Overweight and Obesity
(2021)
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Oleoylethanolamide (oleic monoethanolamide, OEA), the naturally occurring amide of ethanolamine and oleic acid, is an endogenous lipid that modulates feeding, body weight, and lipid metabolism by binding with high affinity to the ligand-activated transcription factor, peroxisome proliferator-activated receptor-alpha (PPAR-alpha). OEA reveals the pharmacological properties in the treatment of obesity, atherosclerosis and other diseases. It was shown, that OEA can be used to control hunger in Prader-Willi syndrome, in addition, it exhibited neuroprotective properties in Parkinson's disease experiments. OEA is an endogenous ligand of the orphan receptor GPR119, a G protein-coupled receptor (GPCR) expressed predominantly in the human and rodent pancreas and gastrointestinal tract and in rodent brain, suggesting that the reported effects of OEA on food intake may be mediated, at least in part, via the GPR119 receptor. Recently was shown, that OEA was an effective inhibitor of hyperpigmentation through activation of ERK, Akt and p38 pathways, inhibition of the CREB pathway, and subsequent down-regulation of MITF, TRP-1 and tyrosinase production. Therefore, OEA could be a useful therapeutic agent for use in the treatment of hyperpigmentation and could be an effective component in whitening and lightening cosmetics.
Status:
US Approved Rx
(2013)
Source:
ANDA200219
(2013)
Source URL:
First marketed in 1928
Class:
PROTEIN
Targets:
Conditions:
More than a century ago, Sir Henry Dale demonstrated that a component of the pituitary causes contractions of the mammalian uterus, hence his coining the term “oxytocic,” derived from the Greek for “quick birth,” for its activity. The discovery that a component of the pituitary causes milk secretion followed within a few years. By 1930, oxytocin was separated from vasopressin into pitocin and pitressin, respectively, at Parke Davis and made available for research. That a single peptide was responsible for these uterine and mammary actions was definitively confirmed upon the sequencing and synthesis of the peptide, 9 amino acids in length. Vincent du Vigneaud was awarded a Nobel Prize for this work.
Oxytocin is indicated for the initiation or improvement of uterine contractions, where this is desirable and considered suitable for reasons of fetal or maternal concern, in order to achieve vaginal delivery. Oxytocin is indicated to produce uterine contractions during the third stage of labor and to control postpartum bleeding or hemorrhage. Uterine motility depends on the formation of the contractile protein actomyosin under the influence of the Ca2+- dependent phosphorylating enzyme myosin light-chain kinase. Oxytocin promotes contractions by increasing the intracellular Ca2+. Oxytocin has specific receptors in the myometrium and the receptor concentration increases greatly during pregnancy, reaching a maximum in early labor at term. The Oxytocin receptor is a typical class I G protein-coupled receptor that is primarily coupled via G(q) proteins to phospholipase C-beta. The high-affinity receptor state requires both Mg(2+) and cholesterol, which probably function as allosteric modulators. The agonist-binding region of the receptor has been characterized by mutagenesis and molecular modeling and is different from the antagonist binding site. The function and physiological regulation of the Oxytocin system is strongly steroid dependent.
Status:
US Approved Rx
(2020)
Source:
ANDA210799
(2020)
Source URL:
First approved in 1973
Source:
HYPERSTAT by SCHERING
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Conditions:
Diazoxide is a drug which was approved by FDA for the treatment of secondary hyperinsulinemia. The drug exerts its action by binding to SUR1 subunit of ATP-sensitive potassium channel that leads to the channel opening.
Status:
US Previously Marketed
Source:
Stop-Zit by Nelson Baker & Company
(1962)
Source URL:
First approved in 1962
Source:
Stop-Zit by Nelson Baker & Company
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Conditions:
Denatonium, usually available as denatonium benzoate (trade names Bitrex) is the most bitter chemical compound known, with bitterness thresholds of 0.05 ppm for the benzoate and 0.01 ppm for the saccharide. Scientists at Macfarlan Smith, Ltd. of Edinburgh, Scotland discovered Bitrex during research on derivatives of the anesthetic lidocaine. The extremely bitter taste proved effective in reducing ingestion by humans and animals. Denatonium is commonly included in placebo medications used in clinical trials to match the bitter taste of certain medications. Denatonium activates bitter taste receptor, mainly, TAS2R4, TAS2R8, TAS2R10, TAS2R13 on many cell types and plays important roles in chemical release, ciliary beating and smooth muscle relaxation through intracellular Ca(2+)-dependent pathways.