200 mg dispersible tablets Carglumic acid
Hyperammonaemia due to N-acetylglutamate synthase primary deficiency
Deficiency of N-acetylglutamate synthase (NAGSD) belongs to the rare inborn errors of N-acetylglutamate synthase(NAGSD) belongs to the rare inborn errorsof metabolism affecting the main pathwayof ammonia detoxification in the ureacycle.The disorder leads to a deficiency of N-acetylglutamate (NAG) which serves as theallosteric activator of the first and rate-limitingenzyme of the urea cycle, carbamoylphosphatesynthetase 1.Carglumic acid, the active substance in Ucedane®is a structural analogue of N-acetylglutamate and mimics its action in patients who cannot synthesize carbamoylphosphate synthetase 1 due to an inherited deficiency in NAGS.
• No special storage conditions
• Has a shelf life of 36 months • Available in blister format (12 tablets per strip)
• Ucedane® is packed in boxes of 12 or 60 tablets
• The tablets are rod-shaped, white and biconvex with three score lines on both sides and engraving “L/L/L /L” on one side.
• The tablet can be divided into four equal doses.
• It is well -established that the key element of the treatment of NAGS deficiency is the total daily dose of carglumic acid. Based on pharmacokinetic characteristics of the product and clinical experience, the product allows some flexibility with regard to the frequency of administration (1). It is recommended to divide the total daily dose into two to four intakes to be taken before meals or feedings. The scoring of the tablet facilitates most of the required posology adjustments.
(1)Carbaglu : EPAR – Scientific Discussion. Nov 2006.