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Diosmin
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DB08995 |
[Diosmin is a semisynthetic drug indicated for the treatment of venous disease. Diosmin is a flavone that can be found in the plant Teucrium gnaphalodes. Diosmin is available as a prescription medicine in several European countries, and is available as a nutritional supplement in the United States and the rest of Europe. It should be noted that clinical studies have been inconclusive and no articles have been published pertaining to its use in the treatment of vascular disease. When used in rats, diosmin has been effective at mitigating hyperglycaemia, and may also have antineurodegenerative properties.] |
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Ingenol mebutate
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DB05013 |
[Ingenol mebutate was approved by the FDA in January 2012, and it is marketed under the name Picato®. Picato gel is indicated for the topical treatment of actinic keratosis. Before approval, ingenol mebutate was called PEP005 as an investigational drug. PEP005 is a selective small molecule activator of protein kinase C (PKC) extracted from the plant Euphorbia peplus, whose sap has been used as a traditional medicine for the treatment of skin conditions including warts and cancer. PEP005 also has potent anti-leukemic effects, inducing apoptosis in myeloid leukemia cell lines and primary AML cells at nanomolar concentrations.] |
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3-({[(3S)-3,4-dihydroxybutyl]oxy}amino)-1H,2'H-2,3'-biindol-2'-one
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DB07676 |
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XEN2174
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DB05012 |
[Xen2174 is a synthetic drug modeled on a peptide from the venom of a cone shell found on Australia's Great Barrier Reef. Xen2174 represents a new class of molecules, called the chi conopeptides that selectively inhibit the Norepinephrine Transporter (NET). NET is the primary mechanism regulating the biological effects of norepinephrine (NE) on the body. In episodes of pain, inhibition of NET by Xen2174 elevates the levels of NE leading to the activation of inhibitory pathways preventing pain signals from reaching the brain. ] |
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Teprotumumab
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DB06343 |
[A fully human IgG1 type monoclonal antibody directed against the human insulin-like growth factor-I receptor.] |
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2-methyl-3,5,7,8-tetrahydro-4H-thiopyrano[4,3-d]pyrimidin-4-one
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DB07677 |
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Taberminogene vadenovec
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DB05011 |
[Taberminogene vadenovec (EG004) is a novel product consisting of a local delivery device and a gene-based medicine, being developed to prevent the blocking of veins and arteries that frequently occurs after vascular surgery. The initial target market is haemodialysis graft access surgery, a procedure in which patients whose kidneys have failed have a plastic tube grafted between blood vessels generally in their forearm so that their blood can be regularly filtered using a dialysis machine. EG004 has completed a Phase II trial, this being the first part of the Phase II/III study for which it has received approval from the US Recombinant DNA Advisory Committee (RAC).] |
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Coltuximab ravtansine
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DB06342 |
[Coltuximab ravtansine (SAR3419) targets CD19 and is being investigated for the treatment of acute lymphoblastic leukemia (ALL).] |
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(9ALPHA,13BETA,17BETA)-2-[(1Z)-BUT-1-EN-1-YL]ESTRA-1,3,5(10)-TRIENE-3,17-DIOL
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DB07678 |
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SGS-742
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DB05010 |
[SGS-742 has been used in trials studying the treatment of Seizures and Metabolic Disease.] |
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(9S,12S)-9-(1-methylethyl)-7,10-dioxo-2-oxa-8,11-diazabicyclo[12.2.2]octadeca-1(16),14,17-triene-12-carboxylic acid
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DB07679 |
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N,4-Dimethyl-N-(1-phenyl-1H-pyrazol-5-yl)benzenesulfonamide
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DB07670 |
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LC16M8
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DB05019 |
[LC16m8 is a next-generation, attenuated smallpox vaccine that is designed to have a better safety profile, yet be equally effective, compared to conventional smallpox vaccines. It is the only attenuated vaccine to be licensed for use in humans to prevent smallpox infection. ] |
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2-[1-METHYLHEXYL]-4,6-DINITROPHENOL
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DB07671 |
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Migalastat
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DB05018 |
[Fabry disease is a rare, progressive genetic disorder characterized by a defective GLA gene that causes a deficiency in the enzyme alpha-Galactosidase A (alpha-Gal A) [FDA Label, F1107, L4274, L4263]. This enzyme is responsible for breaking down glycosphingolipid substrate that, when deficient in patients with Fabry disease, builds up in the blood vessels, the kidneys, the nerves, the heart, and other organs [FDA Label, F1107, L4274, L4278]. In the U.S., it is estimated that more than 3,000 people are living with Fabry disease, and an estimated more than 50 percent of these diagnosed patients are currently untreated [L4274].
Migalastat (approved and sold under Amicus Therapeutics' brand name Galafold) is subsequently an oral pharmacological chaperone of alpha-Gal A for the treatment of Fabry disease in adults who have amenable GLA variants [FDA Label, F1107, L4274, L4278]. In these patients, migalastat works by stabilizing the body’s own dysfunctional alpha-Gal A enzyme so that it can clear the accumulation of glycosphingolipid disease substrate [FDA Label, F1107, L4274, L4278]. Globally, it is estimated that approximately 35 to 50 percent of Fabry patients may have amenable GLA variants that are treatable with migalastat [L4274].
Given the rarity of Fabry disease and the proportion of Fabry disease patients that could benefit from migalastat therapy, Amicus Therapeutics' brand name Galafold was approved using the Accelerated Approval pathway, under which the FDA may approve drugs for serious conditions where there is an unmet medical need and where a drug is shown to have certain effects that are reasonably likely to predict a clinical benefit to patients [FDA Label, F1107, L4274, L4278]. A further study is required to verify and describe the clinical benefits of Galafold, and the sponsor will be conducting a confirmatory clinical trial of Galafold in adults with Fabry disease [FDA Label, F1107, L4274, L4278].
Additionally, Galafold was alzo granted Priority Review designation, under which the FDA’s goal is to take action on an application within six months of application filing where the agency determines that the drug, if approved, would provide a significant improvement in treating, diagnosing or preventing a serious condition over available therapies [FDA Label, F1107]. Galafold also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases [FDA Label, F1107].
As of August 2018, migalastat under Amicus Therapeutics' brand name Galafold is currently approved in Australia, Canada, European Union, Israel, Japan, South Korea, Switzerland, and the United States.] |
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TRANS-2-(DIMETHYLPHENYLSILYL)-PIPERIDINE-N-OXIDE
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DB07672 |
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YSIL6
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DB05017 |
[YSIL6 is a small-molecule drug in development for the treatment of inflammatory diseases, including rheumatoid arthritis and psoriasis. The molecule works by inhibiting TNF-alpha and IL-6 production in T-cells and macrophages, and by inhibiting T-cell proliferation and migration.] |
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DEAMINO-METHYL-PHENYLALANINE
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DB07673 |
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Cenisertib
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DB06347 |
[Cenisertib is an aurora kinase inhibitor.] |
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Ataluren
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DB05016 |
[Ataluren is a novel, orally administered drug that targets nonsense mutations. Ataluren is approved for use by the European Medicines Agency to treat Duchenne Muscular Dystrophy in patients aged 5 years and older who are able to walk. More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
This drug does not yet have approval by the US Food and Drug Administration or by Health Canada for any indications.] |