{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
{{facet.count}}
Search results for "ORPHAN DRUG" in comments (approximate match)
Status:
Possibly Marketed Outside US
First approved in 2017
Source:
M020
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Conditions:
Cinnamic acid is a polyphenol found in cinnamon oil and used in commercial flavorings. Recent studies have shown the pharmacological properties of cinnamic acid and its derivatives, including hepatoprotective, anti-oxidant, and anti-diabetic activities. In preclinical studies cinnamic acid demonstrated to be a promising candidate for the treatment ob obesity and diabetes. The mechanism of action of cinnamic acid in obesity is explained by its ability to inhibit lipases and ACE (angiotensin-converting enzyme). However, there are several hypotesis regarding the effect of cinnamic acid in diabetes: cinnamic acid enhances glucose-induced insulin secretion, prevents palmitic acid-induced lipotoxicity, inhibits palmitic acid-induced alteration of lipogenic gene and protein expression (AMPK, SREBP-1c, FAS, ACC), inhibits DPP IV, exhibits an additive effect on the uptake of glucose, stimulates adiponectin secretion, etc.
Status:
Possibly Marketed Outside US
Source:
NDA213051
(2017)
Source URL:
First approved in 2017
Source:
NDA213051
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Salcaprozate sodium (SNAC), an oral absorption promoter that was discovered as part of a screen to identify carrier-based permeation enhancers (Pes) that could “chaperone” poorly permeable payloads across the intestine. Its potential therapeutic application as a delivery agent was tested in many formats: taste-masked liquids, tablets, and soft gelatin capsules. SNAC is the most extensively tested carrier and the only PE approved in an oral formulation designed to improve oral bioavailabilities. The mechanism of action of this compound is not clear. However, Novo Nordisk offered a mechanism of action for SNAC in its non-enteric coated tablet of the glucagon-like peptide 1 analog, semaglutide. SNAC formed a complex around the semaglutide in the stomach and caused a transient increase in local pH around the molecule. It is claimed that semaglutide is protected against pepsin by SNAC and that solubility was increased, resulting in an increased concentration-dependent flux of semaglutide across the gastric mucosa, using a transcellular mechanism as the tablet comes in intimate contact with the epithelium. Clinical trials for patients with Type 2 Diabetes have shown that the oral semaglutide co-formulated with 300 mg SNAC could be used for further clinical development.
Status:
Possibly Marketed Outside US
Source:
CARBOSAN by Brown, H.M. et al.
Source URL:
First approved in 2016
Source:
21 CFR 347
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
Carbenoxolone is a glycyrrhetinic acid derivative with a steroid-like structure, similar to substances found in the flavor-ful root of the licorice plant. It influences endogenous glucocorticoids by potently inhibiting 11β-hydroxysteroid dehydrogenase. Electrolyte imbalance is a serious side effect of carbenoxolone when used systemically. Carbenoxolone is best known in cellular physiology as a modestly potent, reasonably effective, water-soluble blocker of gap junctions. It exerts anti-inflammatory activity. Carbenoxolone has used orally in the clinical treatment of peptic ulcers, now it is used topically for the treatment of lip sores and mouth ulcers.
Status:
Possibly Marketed Outside US
Source:
21 CFR 347
(2016)
Source URL:
First approved in 2016
Source:
21 CFR 347
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
Lactic acid, D- is a natural optical isomer of lactic acid. It is a poorly utilized isomer – 30 to 40% of the dose ingested is excreted in the urine. Lactic acid, D- is known to be harmful to human metabolism and it can result in acidosis and decalcification. D-lactic acidosis, also referred as D-lactate encephalopathy, has been reported in patients with short bowl syndrome. Lactic acid, D- is an interesting precursor for manufacturing heat-resistant polylactic acid (PLA) bioplastics which can be widely used, for example as packaging material, coatings, for textiles or in the automotive industry.
Status:
Possibly Marketed Outside US
Source:
M020
(2015)
Source URL:
First approved in 2015
Source:
M020
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Conditions:
Oxothiazolidinecarboxylic Acid (L-2-oxothiazolidine-4-carboxylic acid, OTC) was currently undergoing evaluation in a Phase II clinical trial. It is suggested that an ideal drug may be an antisense oligonucleotide that blocks the expression of glycolate oxidase, a key enzyme in hepatic oxalate synthesis.
Status:
Possibly Marketed Outside US
First approved in 2015
Source:
Co-Balamin by Home Aide Diagnostics, Inc.
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
UBIQUINOL is a reduced form of coenzyme Q10, a lipid-soluble molecule that consists of an aromatic ring and a 10-unit isoprenoid chain. It is naturally produced by the body. A significant increase in plasma CoQ10 status observed after supplementation in one study suggested that UBIQUINOL is a better supplemental form to enhance the CoQ10 status than ubiquinone (the oxidized form of Q10). UBIQUINOL has also been found to reduce oxidative stress induced by strenuous exercise. Oxidative stress is associated with aging and many chronic diseases, such as cardiovascular diseases, cancers, and cognitive decline. Ubiquinol has furthermore been shown to improve mitochondrial oxidative metabolism in the brain, and cognitive improvement was found as a result of Ubiquinol intake in chronic fatigue syndrome patients. UBIQUINOL is considered safe, but can have side effects such as upper abdominal pain, loss of appetite, nausea, diarrhea and headaches. CoQ10 might make blood-thinning drugs, such as warfarin (Coumadin, Jantoven), less effective. This could increase the risk of a blood clot.
Status:
Possibly Marketed Outside US
Source:
21 CFR 348
(2018)
Source URL:
First approved in 2015
Source:
21 CFR 348
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
LAUROCAPRAM is a percutaneous permeation enhancer facilitating absorption of different chemicals upon their application to the skin. Its enhancing effect is associated with different mechanisms: incorporation of its dodecyl group into the lipid bilayer, an increase of the motion of the alkylic chains of lipids, and lipid fluidization.
Status:
Possibly Marketed Outside US
Source:
21 CFR 352
(2017)
Source URL:
First approved in 2014
Source:
21 CFR 352
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Status:
Possibly Marketed Outside US
Source:
Dr. Cellapy SR Premium Solution by GM Holdings Co., Ltd
(2014)
Source URL:
First approved in 2014
Source:
Dr. Cellapy SR Premium Solution by GM Holdings Co., Ltd
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Conditions:
Cytosine is a pyrimidine nucleobase, one of the five main bases of nucleic acids. In DNA and RNA cytosine is paired with guanine. Only small amounts of cytosine administered with food are incorporated in DNA. The majority of cytosine is synthesized de-novo starting from carbamoyl phosphate.
Status:
Possibly Marketed Outside US
Source:
ROBENGATOPE by Photogen Technologies
Source URL:
First approved in 2013
Source:
GloStrips by Nomax Inc.
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Rose bengal sodium (RB) is a small molecule, halogenated xanthene being developed by Provectus Biopharmaceuticals (formerly Provectus Pharmaceuticals). It is commonly used in eye drops to stain damaged conjunctival and corneal cells and thereby identify damage to the eye. The stain is also used in the preparation of Foraminifera for microscopic analysis, allowing the distinction between forms that were alive or dead at the time of collection. A form of rose bengal is also being studied as a treatment for certain cancers and skin conditions. The cancer formulation of the drug, known as PV-10, is currently undergoing clinical trials for melanoma and breast cancer. Recently, interest in RB as a therapeutic cancer treatment has increased due to significant anti-tumor responses with direct tumor injection in human clinical trials for metastatic melanoma. In these patients, there has been the implication that RB may mount a T-cell mediated anti-tumor response and impart antigen-specific responses in distant bystander lesions.