The Organic Acidemia drugs in development market research report provides comprehensive information on the therapeutics under development for Organic Acidemia, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA), and molecule type. GlobalData’s report assesses key aspects of the companies and drugs in development for Organic Acidemia. Buy the report here.

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Data Insights Organic Acidemia - Drugs In Development, 2024

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The report also covers the descriptive pharmacological action of the therapeutics and the latest news and press releases. Additionally, the report provides an overview of the key players involved in therapeutic development for Organic Acidemia and features dormant and discontinued products.

GlobalData tracks five drugs in development for Organic Acidemia by four companies/universities/institutes. The top development phase for Organic Acidemia is preclinical with four drugs in that stage. The Organic Acidemia pipeline has five drugs in development by companies and 0 by universities/ institutes. Some of the companies in the Organic Acidemia pipeline products market are: Synlogic, Icagen and Ironwood Pharmaceuticals.

The key targets in the Organic Acidemia pipeline products market include Branched Chain Amino Acid Aminotransferase Mitochondrial.

The key mechanisms of action in the Organic Acidemia pipeline product include Branched Chain Amino Acid Aminotransferase Mitochondrial Activator with one drug in Preclinical. The Organic Acidemia pipeline products include two routes of administration with the top ROA being Oral and two key molecule types in the Organic Acidemia pipeline products market including Small Molecule, and Biologic.

Organic Acidemia overview

Organic acidemia or organic acidurias are a group of inherited metabolic disorders characterized by the accumulation of abnormal organic acid metabolites in the body fluids and tissues. They are caused by a deficiency in an enzyme necessary to break down amino acids, thus causing increased organic acids in body fluids that are then excreted in urine. Vomiting, poor feeding, failure to thrive, hypoglycemia, hyperammonemia, seizures, hypotonia, lethargy, coma, apnea, and respiratory distress are the symptoms of organic acidemia. The most common test for diagnosing organic acidemia is urine organic acid analysis. Levocarnitine and glycine medications, a restricted protein diet and amino acid supplements, and increasing calories and fluids are treatment options.

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GlobalData’s pipeline drugs offers detailed profiles of pharmaceutical drugs in all stages of pre-clinical and clinical development, from discovery through to pre-registration. Coverage is limited to novel human medicinal drugs and biosimilars seeking market approval proprietary and is one of two primary repositories of pharmaceutical drug information offered by GlobalData through its Pharmaceutical Intelligence Center.