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Volixibat
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Looking for Volixibat API 1025216-57-2?
- Description:
- Here you will find a list of producers, manufacturers and distributors of Volixibat. You can filter on certificates such as GMP, FDA, CEP, Written Confirmation and more. Send inquiries for free and get in direct contact with the supplier of your choice.
- API | Excipient name:
- Volixibat
- Synonyms:
- Cas Number:
- 1025216-57-2
- DrugBank number:
- DB13914
- Unique Ingredient Identifier:
- X2JZ0451H8
General Description:
Volixibat, identified by CAS number 1025216-57-2, is a notable compound with significant therapeutic applications. Volixibat, also known as SHP626 or LUM002, is an investigational drug that will potentially be used for the treatment of Non-Alcoholic Steatohepatitis (NASH). If approved for use, it will be the first available agent for the treatment of NASH. Volixibat is a selective inhibitor of the apical sodium-dependent bile acid transporter (ASBT), a transmembrane protein primarily expressed by enterocytes of the ileum. Also known as the ileal bile acid transporter (IBAT), ASBT is primarily responsible for the enterohepatic recirculation of bile acids and ultimately for hepatic lipid and glucose metabolism . Inhibiting this enzyme results in a decrease of bile acids returning to the liver, which is helpful for the treatment of NASH as abnormal cholesterol metabolism and accumulation of free cholesterol in the liver have been implicated in its pathogenesis. According to Shire, the pharmaceutical manufacturer of Volixibat, it has been granted fast track status by the Food and Drug Administration due to promising initial results and a need for therapeutic treatments for NASH.
Indications:
This drug is primarily indicated for: Volixibat is an investigational drug that has not been approved for use in any conditions. Its use in specific medical scenarios underscores its importance in the therapeutic landscape.
Mechanism of Action:
Volixibat functions by: Volixibat is a selective inhibitor of the apical sodium-dependent bile acid transporter (ASBT), a transmembrane protein primarily expressed by enterocytes of the ileum. ASBT is primarily responsible for the recirculation of bile acids and therefore for hepatic lipid and glucose metabolism. Inhibiting this enzyme results in a decrease of bile acids returning to the liver, which is helpful for the treatment of NASH as abnormal cholesterol metabolism and accumulation of free cholesterol in the liver have been implicated in its pathogenesis. This mechanism highlights the drug's role in inhibiting or promoting specific biological pathways, contributing to its therapeutic effects.
Classification:
Volixibat belongs to the class of organic compounds known as hexoses. These are monosaccharides in which the sugar unit is a is a six-carbon containing moeity, classified under the direct parent group Hexoses. This compound is a part of the Organic compounds, falling under the Organic oxygen compounds superclass, and categorized within the Organooxygen compounds class, specifically within the Carbohydrates and carbohydrate conjugates subclass.
Categories:
Volixibat is categorized under the following therapeutic classes: Carbohydrates, Heterocyclic Compounds, Fused-Ring, Lipid Regulating Agents, Sulfur Compounds. These classifications highlight the drug's diverse therapeutic applications and its importance in treating various conditions.
Volixibat is a type of Enzyme Replacements/modifiers
Enzyme replacements/modifiers are a crucial category of pharmaceutical active pharmaceutical ingredients (APIs) utilized in the treatment of various enzyme-related disorders. Enzymes play a vital role in the normal functioning of the body by catalyzing specific biochemical reactions. However, in certain medical conditions, the body may lack or produce dysfunctional enzymes, leading to serious health complications.
Enzyme replacement therapy (ERT) involves administering exogenous enzymes to compensate for the enzyme deficiency in patients. These enzymes are typically derived from natural sources or produced using recombinant DNA technology. By introducing these enzymes into the body, they can effectively substitute the missing or defective enzymes, thereby restoring normal metabolic processes.
On the other hand, enzyme modifiers are API substances that regulate the activity of specific enzymes within the body. These modifiers can either enhance or inhibit the enzyme's function, depending on the therapeutic objective. By modulating enzyme activity, these APIs can restore the balance of enzymatic reactions, leading to improved physiological outcomes.
Enzyme replacements/modifiers have shown remarkable success in treating various genetic disorders, such as Gaucher disease, Fabry disease, and lysosomal storage disorders. Additionally, they have demonstrated potential in managing enzyme deficiencies associated with rare diseases and certain types of cancer.
The development and production of enzyme replacements/modifiers involve rigorous research, formulation optimization, and adherence to stringent quality control measures. Pharmaceutical companies invest substantial resources in developing these APIs to ensure their safety, efficacy, and compliance with regulatory standards.
Overall, enzyme replacements/modifiers represent a vital therapeutic category in modern medicine, offering hope and improved quality of life for patients with enzyme-related disorders.