Carglumic Acid

Congenital hyperammonemia is a fatal metabolic abnormality caused by urea cycle disorders, and there has been a lack of targeted long-term intervention solutions for a long time. The advent of carglumic acid has filled this clinical gap. As a structural analog of N-acetylglutamate (NAG), it belongs to the class of urea cycle activating drugs. It can restart the urea cycle pathway by activating carbamoyl phosphate synthetase 1 in the liver, and promote the metabolic clearance of ammonia in the body. At present, this drug is mainly used for the treatment of acute hyperammonemia caused by N-acetylglutamate synthetase deficiency, propionic acidemia or methylmalonic acidemia in neonates and adults, as well as long-term maintenance treatment. It is a key therapeutic drug in the field of rare disease metabolism.

The current global market size of carglumic acid is approximately USD 180 million, and the compound annual growth rate in the past three years has maintained at around 7.2%. The core growth drivers come from the implementation of rare disease security policies in various countries and the increase in the screening rate of urea cycle disorders. Due to the niche clinical demand and high barriers to synthesis technology of this drug, the market competition pattern is highly concentrated. The original research enterprise occupies nearly 90% of the global market share. At present, only 2 domestic enterprises have submitted marketing applications for generic preparations, and there is still a large space for domestic substitution in the supply of active pharmaceutical ingredients.

The original research enterprise of carglumic acid is Orphan Europe SARL, and the original research brand name is Carbaglu. Its compound patent in the European Union expired in 2019, and the drug patent in the United States expired in 2022. The main dosage form approved for the original research product is tablet, with a specification of 200 mg per tablet. It has been included in the FDA Reference Listed Drug Catalog and China's *Catalog of Reference Preparations for Chemical Drugs (Third Batch)*. As of now, no approved A status registration number of carglumic acid has been found on the API registration platform of China's CDE. The original research imported preparation has been approved for marketing in China, but no domestic generic preparation has been approved yet. (Data as of June 2025, please refer to the official CDE website for the latest information)

CATO provides a full set of impurity reference standards for carglumic acid, which can cover the R&D requirements of the whole process of process research, quality research and stability investigation. Most of the products are in stock. Orders for in-stock products placed before 16:00 will be shipped on the same day. Meanwhile, the products meet the compliance requirements of multiple regulations such as the Chinese Pharmacopoeia and FDA, which can help R&D enterprises effectively reduce the cost of impurity research and shorten the project declaration cycle.