Carglumic acid, a new drug, has been registered

Moscow, October 2023

The drug contains an active substance – carglumic acid, which can quickly eliminate hyperammonemia (increased ammonia levels in the blood). Ammonia is particularly toxic to the brain and in severe cases leads to decreased level of consciousness and coma. Hyperammonemia can be caused by:

• Lack of the specific hepatic enzyme N-acetylglutamate synthase. Patients with this rare disorder are unable to excrete nitrogenous wastes that accumulate after protein consumption. This disorder persists throughout the life of the affected patient and therefore the need for this treatment is lifelong.
• Impaired excretion of organic acids, resulting in acidemia – elevated levels of acids in the blood (isovalerian, methylmalonic or propionic acidemia). Patients suffering from one of these disorders require treatment during a crisis of hyperammonemia.

Carglumic acid belongs to the pharmacotherapeutic group "other agents for the treatment of gastrointestinal and metabolic disorders; amino acids and their derivatives".

Note:

The press release contains information about prescription medications. Use of this information for preparation of publications must be made in accordance with the legislation of the Russian Federation on advertising (including but not limited to Art. 24 of the Federal Law of 13.03.2006 No. 38-FL "On Advertising"), according to which, in particular, advertising of prescription drugs is allowed only in places of medical or pharmaceutical exhibitions, workshops, conferences and other similar events as well as in specialized print media intended for medical and pharmaceutical workers. Failure to comply with the above requirements (namely, mentioning the name of the drug, placing an image of the package or its elements in the media other than specialized print publications intended for medical and pharmaceutical workers) shall entail administrative liability.