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Search results for "EU ORPHAN DRUG" in comments (approximate match)
Status:
Possibly Marketed Outside US
Source:
Lipovite by Perdido Key Health And Wellness Inc
Source URL:
First approved in 2012
Source:
SPAI-SONSPROLAC-VIT by SPAI-SONS PHARMACEUTICAL INTERNATIONAL COSMETICS
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Citrulline (name derived from citrullus, the Latin word for watermelon, from which it was first isolated) is an amino acid. It is made from ornithine and carbamoyl phosphate in one of the central reactions in the urea cycle. It is also produced from arginine as a by-product of the reaction catalyzed by nitric oxide synthase (NOS) family. Citrulline supplements have been claimed to promote energy levels, stimulate the immune system and help detoxify ammonia (a cell toxin). Citrulline is not involved in protein synthesis. Several pharmacokinetic studies have confirmed that citrulline is efficiently absorbed when administered orally. Oral citrulline could be used to deliver arginine to the systemic circulation or as a protein anabolic agent in specific clinical situations (for example in case of malnourishment), because recent data have suggested that citrulline, although not a component of proteins, stimulates protein synthesis in skeletal muscle through the mammalian target of rapamycin signaling pathway. Citrulline is converted to L-arginine by argininosuccinate synthase. L-arginine is in turn responsible for citrulline's therapeutic effects. Many of L-arginine's activities, including its possible anti-atherogenic actions, may be accounted for by its role as the precursor to nitric oxide (NO).
Status:
Possibly Marketed Outside US
Source:
NCT03384329: Phase 4 Interventional Completed Depression
(2018)
Source URL:
First approved in 2011
Source:
21 CFR 352
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Resveratrol, a natural non-flavonoid polyphenol, exhibits a wide range of beneficial properties as an anticancer agent, a platelet anti-aggregation agent, and an antioxidant, as well as its anti-aging, anti-inflammatory, antiallergenic. This compound is in phase III clinical trials in combination with carboxymethyl-β-glucan for improving nasal symptoms in children with pollen-induced allergic rhinitis. Also in phase III clinical trial in the treatment of painful knee osteoarthritis and in type 2 diabetic patients. It has been demonstrated that resveratrol may prevent type 2 diabetic by targeting Sirtuin type 1 (SIRT1), indicating that SIRT1 may be a novel therapeutic target for diabetes prevention.
Status:
Possibly Marketed Outside US
Source:
NCT02774005: Phase 4 Interventional Completed Leber's Hereditary Optic Neuropathy (LHON)
(2016)
Source URL:
First approved in 2011
Source:
21 CFR 352
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Conditions:
Idebenone is a synthetic short-chain benzoquinone and a substrate for the enzyme NAD(P)H:quinone oxidoreductase (NQO1) capable of stimulating mitochondrial electron transport and supplementing cellular energy levels. Idebenone was initially developed by Takeda Pharmaceutical Company for the treatment of Alzheimer’s disease and other cognitive defects. This has been met with limited success. The Swiss company Santhera Pharmaceuticals has started to investigate it for the treatment of neuromuscular diseases. In 2010, early clinical trials for the treatment of Friedreich’s ataxia and Duchenne muscular dystrophy have been completed. In clinical trials, Idebenone (Raxone/Catena) had a positive impact on a measurement of respiratory function (Peak Expiratory Flow, or PEF) in non-ambulatory Duchenne muscular dystrophy patients who were not taking steroids. As of December 2013 the drug is not approved for these indications in North America or Europe. It is approved for the treatment of Leber's hereditary optic neuropathy (LHON) in Europe. Idebenone (Raxone) is indicated for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON). Because the number of patients with Leber's hereditary optic neuropathy is low, the disease is considered ‘rare’, and Raxone was designated an ‘orphan medicine’ on 15 February 2007. Idebenone is thought to help improve production of energy by restoring mitochondrial function, thereby preventing the cellular damage and the loss of sight seen in LHON. Idebenone is a rapidly absorbed, safe and well-tolerated drug and is currently the only clinically proven treatment option for Leber's hereditary optic neuropathy (LHON) patients.
Status:
Possibly Marketed Outside US
Source:
NCT00726713: Phase 4 Interventional Completed Type 2 Diabetic Peripheral Neuropathy (DPN)
(2008)
Source URL:
First approved in 2011
Source:
EnLyte by Jaymac Pharma
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Pyridoxal phosphate (PLP, pyridoxal 5'-phosphate, P5P) is a coenzyme, the active form of vitamin B6. Pyridoxal 5′-phosphate (PLP) is used as a cofactor for a wide range of enzymes including mitochondrial cysteine desulfurase, cystathionine γ-synthase (CGS), ornithine 4,5-aminomutase (OAM), and d-serine dehydratase. The versatility of PLP arises from its ability to covalently bind the substrate, and then to act as an electrophilic catalyst, thereby stabilizing different types of carbanionic reaction intermediates. PLP acts as a coenzyme in all transamination reactions, in various beta-elimination reactions, in the condensation reaction in heme synthesis.
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
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.
Status:
Possibly Marketed Outside US
Source:
NCT03626298: Phase 4 Interventional Completed Acne Vulgaris
(2016)
Source URL:
First approved in 2002
Source:
M006
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Conditions:
Zinc Pidolate (Zinc PCA) is a topical skin product with purifying, astingent, anti-inflammatory, antiseptic activity. It has long been used as a cosmetic ingredient, because of its astringent and anti-microbial properties. Zinc Pidolate has also being shown to be effective against halitosis. Zinc PCA prevents the UV-induced MMP-1 production in vitro by suppressing the activation of AP-1. Zinc PCA was also able to enhance type I collagen synthesis in NHDFs, by increasing the expression of the mRNA encoding the ascorbic acid transporter SVCT2 in non-UV irradiated
NHDFs, which suggests its promising effect against not only photoaged skin but also for the simple atrophic change of intrinsic skin ageing. Zinc PCA is able to suppress sebum secretion by inhibiting 5-α reductase in hyperseborrhea, to suppress body odor by forming zinc salts with short-chain fatty acids, to suppress wrinkles by inhibiting AP-1 to and inhibit bacterial growth including acne related Propionibacterium acnes.
Status:
Possibly Marketed Outside US
Source:
NCT04475276: Phase 4 Interventional Recruiting Non-Alcoholic Fatty Liver Disease
(2021)
Source URL:
First approved in 2001
Source:
Strovite OneCaplets by Exeltis USA, Inc.
Source URL:
Class (Stereo):
CHEMICAL (RACEMIC)
Conditions:
Thioctic acid also known as alpha-lipoic acid is a dietary supplement, which is a common ingredient in OTC (over-the-counter) multivitamin formulas and anti-aging supplements. Thioctic acid exists in both R- and S-enantiomeric forms, however, only R-form is essential as a cofactor in biological systems (the acid is coupled via an amide linkage to a lysine of several multienzyme complexes, such as the pyruvate dehydrogenase complex, the alpha-ketoglutarate dehydrogenase complex, the glycine cleavage system and the branched-chain oxo acid dehydrogenase complex). Most commercially available thioctic acid supplements are a mixture of both R and S enantiomers or R-form alone. Several studies have shown that the acid has beneficial effect on diabetes complications, cancer, glaucome, liver disease, etc. The mechanisms of thioctic acid is related to its antioxidant properties, metal chelator properties, however, those mechanisms need futher confirmation.
Status:
Possibly Marketed Outside US
Source:
NCT02644707: Phase 4 Interventional Completed Autoimmune Thyroiditis
(2014)
Source URL:
First approved in 2000
Source:
BACMIN by Marnel Pharmaceuticals, Inc.
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Conditions:
L-isomer of selenomethionine (Se-met) is a major natural food-form of selenium, synthetic L-Se-met or enriched food sources thereof such as selenium yeast are appropriate supplemental forms of Se for humans; for animals, DL-Se-met is acceptable. Ingested Se-met is either metabolized directly to reactive forms of selenium or stored in place of methionine in body proteins. Se-met metabolism is closely linked to protein turnover. Selenium, which is nutritionally essential for humans, is a constituent of more than two dozen selenoproteins that play critical roles in reproduction, thyroid hormone metabolism, DNA synthesis, and protection from oxidative damage and infection.
Status:
Possibly Marketed Outside US
Source:
NDA020657
(1997)
Source URL:
First approved in 1997
Source:
NDA020657
Source URL:
Class (Stereo):
CHEMICAL (MIXED)