Carglumic acid is an orphan drug that is utilized for treatment of patients with hyperammonemia, which implies deficiency of N-acetylglutamate (NAG) synthase. Well known as N-carbamoyl-L-glutamic acid (NCGA) or Carbaglu, carglumic acid stimulates the initial enzyme in urea cycle, which helps in detoxification and removal of ammonia from blood cells. Carglumic acid is on the verge of becoming an indispensable therapeutic tool for managing life-threating & rare inborn metabolic disorders that affect the urea cycle.
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Carglumic Acid holds Potential in Accelerating Cancer Cell Suppression and Promoting Apoptosis
Analysis of in vivo models have showcased carglumic acid to stimulate liver carbamoyl phosphate synthetase. Although carbamoyl phosphate synthetase has a relatively lower affinity for carglumic acid than N-acetylglutamate (NAG), carglumic acid has been represented in vitro to activate carbamoyl phosphate synthetase, being much more effective in preventing ammonia intoxication. This orphan drug is currently gaining huge traction in treating patients with hyperammonemia, a rare congenital disorder caused by genetic defects pertaining to urea cycle enzymes. Carglumic acid also holds potential in accelerating cancer cell suppression, thereby promoting apoptosis in various types of cancers such as lung cancer and pancreatic ductal adenocarcinoma. These Factors will determine the future expansion of the market for carglumic acid across the globe.
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Researchers across the globe are working on the development of “whole exome sequencing” procedure, with an aim of preventing intellectual disability in children through the combination of metabolomics analysis of carglumic acid and genome sequencing. Carglumic acid is further being utilized for application in enhancing ammonia detoxification in patients with organic acidurias. Transparency Market Research’s new report states that the global carglumic acid market will reflect an impressive expansion through the forecast period (2017-2026). Worldwide sales of carglumic acid is estimated to reach revenues worth nearly US$ 190 Mn by 2026-end.
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