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Search results for "ORPHAN DRUG|Designated|Treatment of Amyotrophic lateral sclerosis" in comments (approximate match)
Status:
US Previously Marketed
Source:
21 CFR 310.545(a)(20) weight control isoleucine
Source URL:
First approved in 1971
Source:
NDA016822
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Isoleucine is an essential α-amino acid that is used in the biosynthesis of proteins. L-isoleucine is commonly used in parenteral and enteral nutrition. It is used in combination with the other branched chain amino acids to improve the nutritional status of patients with hepatic diseases. BCAAs serve as important fuel sources for skeletal muscle during periods of metabolic stress
Status:
US Previously Marketed
Source:
21 CFR 310.545(a)(20) weight control valine
Source URL:
First approved in 1971
Source:
NDA016822
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Conditions:
Valine is an α-amino acid that is used in the biosynthesis of proteins. It is essential in humans, meaning the body cannot synthesize it and thus it must be obtained from the diet. There is a mixed scientivic evidence that as a dietary supplement valine improves training efficacy, delays central fatigue.
Status:
US Previously Marketed
Source:
glycolic acid
(2022)
Source URL:
First approved in 1947
Source:
NDA006188
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Conditions:
SODIUM GLYCOLATE, a sodium salt of carboxymethyl ether, is used as a pharmaceutical grade dissolution excipient for solid dosage forms. It absorbs water rapidly, resulting in swelling which leads to the rapid disintegration of tablets and capsules. Without a disintegrant, these may not dissolve appropriately and may effect the amount of active ingredient absorbed, thereby decreasing effectiveness.
Status:
US Previously Marketed
Source:
Dinitrophenol
(1933)
Source URL:
First marketed in 1933
Class (Stereo):
CHEMICAL (ACHIRAL)
Conditions:
2,4-dinitrophenol (DNP) is a FDA-banned weight-loss agent and EPA-regulated environmental toxicant, traditionally used in research labs as an uncoupler of OXPHOS. Although not licensed for human consumption, DNP and DNP crystal form are used by bodybuilders and extreme dieters for their fat burning properties through inhibiting efficient energy (ATP) production in cells. Through uncoupling mitochondrial oxidative phosphorylation by facilitating proton transport across the mitochondrial membrane, DNP leads to rapid consumption of energy without generating ATP and consequently, to increased fat metabolism. However, the weight-loss effect comes with serious, and in some cases potentially fatal, adverse side effects, namely hyperthermia (the leading cause of fatality with acute DNP toxicity) and cardiac arrest, but also diaphoresis, tachycardia, tachypnea, skin toxicity, Fourier’s gangrene and cataracts with low dose chronic exposure. The proposed mechanism of DNP induced toxicity suggests the activation of ATP-sensitive K channels.
Status:
US Previously Marketed
Source:
Narcotine by Merck
(1897)
Source URL:
First marketed in 1897
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Noscapine (also known as Narcotine, Nectodon, Nospen, Anarcotine and (archaic) Opiane) is a benzylisoquinoline alkaloid from plants of the poppy family, without painkilling properties. This agent is primarily used for its antitussive (cough-suppressing) effects. Noscapine is often used as an antitussive medication. A 2012 Dutch guideline, however, does not recommend its use for coughing. Noscapine can increase the effects of centrally sedating substances such as alcohol and hypnotics. Noscapine should not be taken in conjunction with warfarin as the anticoagulant effects of warfarin may be increased. Noscapine, and its synthetic derivatives called noscapinoids, are known to interact with microtubules and inhibit cancer cell proliferation. Mechanisms for its antitussive action are unknown, although animal studies have suggested central nervous system as a site of action. Furthermore, noscapine causes apoptosis in many cell types and has potent antitumor activity against solid murine lymphoid tumors (even when the drug was administered orally) and against human breast and bladder tumors implanted in nude mice. Because noscapine is water-soluble and absorbed after oral administration, its chemotherapeutic potential in human cancer merits thorough evaluation. Antifibrotic effect of noscapine based on novel mechanism, which it shows through EP2 prostaglandin E2 receptor-mediated activation of protein kinase A.
Status:
Possibly Marketed Outside US
Source:
M017
(2024)
Source URL:
First approved in 2024
Source:
M017
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Oxaloacetate (OAA), a salt of oxaloacetic acid, is a metabolic intermediate in many processes, e.g., urea cycle, gluconeogenesis, etc. that occur in animals. Experiments on animal have revealed that OAA was able to protect hepatocytes from hypoxia and liver ischemia/reperfusion injury. OAA also possesses a neuroprotective effect against ischemic injury, which strengthens the likelihood of its future applicability as a novel neuroprotective agent for the treatment of ischemic stroke patients. In addition, experiments on adipose stromal cells have shown that OAA directly protected cerebellar granule neurons from apoptosis induced by serum and potassium deprivation.
Status:
Possibly Marketed Outside US
Source:
Sexual Function Activator by Sanaura Group LIMITED
(2024)
Source URL:
First approved in 2024
Source:
Sexual Function Activator by Sanaura Group LIMITED
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Smilagenin (also known as PYM50028 and Cogane) is a constituent of Jamaican sarsaparilla (Smilax ornata) and is a neuroprotectant that has been evaluated in Phase II clinical trials in Parkinson's disease and Alzheimer's disease. On 27 September 2011, the European Commission to Phytopharm plc, United Kingdom, granted orphan designation for smilagenin for the treatment of amyotrophic lateral sclerosis. The details of the mechanism of action of smilagenin are incomplete but involve the upregulation of neurotrophic factors including Glial cell line-derived neurotrophic factor and brain-derived neurotrophic factor.
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
Source:
CLENBUTEROL by Boehringer Ingelheim
Source URL:
First approved in 2011
Source:
NADA140973
Source URL:
Class (Stereo):
CHEMICAL (RACEMIC)
Targets:
Clenbuterol is agonist of beta2 adrenergic receptor. In some countries it is used as bronchodilator for treatment of asthma, but is not approved in USA. The drug is abused by bodybuilders and athletes for its ability to increase lean muscle mass and to reduce body fat. In 1998 FDA approved the clenbuterol-based Ventipulmin Syrup as a prescription-only drug for treatment of airway obstruction in horses.