Carglumic acid in hyperammonaemia due to organic acidurias: a profile of its use in the EU
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Carglumic acid (N-carbamylglutamate; Carbaglu®), a structural analog of N-acetylglutamate, is an effective and generally well-tolerated option for the treatment of hyperammonaemia due to propionic, methylmalonic and isovaleric acidurias. These organic acidurias are linked to a group of autosomal recessive inherited disorders that are associated with secondary hyperammonaemia due to the inhibition of the urea cycle. Carglumic acid can be administered orally or through a nasogastric tube. In retrospective, observational studies and case series/reports, carglumic acid rapidly reduced plasma ammonia levels and improved clinical symptoms of hyperammonaemia. Carglumic acid is generally well tolerated, and, based on limited data, may be associated with fewer treatment-emergent adverse events when used in combination with ammonia scavengers than when either therapy is administered alone.
The manuscript was reviewed by: A. Chakrapani, Metabolic Medicine Department, Great Ormond Street Hospital, NHS Foundation Trust, London, UK; S. Yap, Department of Inherited Metabolic Disorders, Sheffield Children’s Hospital, NHS Foundation Trust, Sheffield, UK. During the peer review process, Orphan Europe–Recordati Group, the marketing-authorization holder of carglumic acid, was also offered an opportunity to provide a scientific accuracy review of their data. Changes resulting from comments received were made on the basis of scientific and editorial merit.
Compliance with ethical standards
The preparation of this review was not supported by any external funding.
Conflict of interest
H A. Blair is an employee of Adis/Springer, is responsible for the article content and declares no conflicts of interest.
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