SAMOH Pharm CO., LTD. & SAMOH Pharmachem CO., LTD.

Indication/Usage 

[Indication] 

hyperammonaemia due to NAGS(N-acetylglutamate synthase primary deficiency), or isovaleric acidaemia, or methymalonic acidaemia, or propionic acidaemia. 

[Dosage and Method of Administration] 

Carbaglu treatment should be initiated under the supervision of a physician experienced in the treatment of metabolic disorders. 

1. Posology 

A. For N-acetylglutamate synthase deficiency 

The treatment may be started as early as the first day of life. 

The initial daily dose should be 100 mg/kg, up to 250 mg/kg if necessary. 

It should then be adjusted individually in order to maintain normal ammonia plasma levels. 

In the long term, it may not be necessary to increase the dose according to body weight as long as adequate metabolic control is achieved; daily doses range from 10 mg/kg to 100 mg/kg. 

Carglumic acid responsiveness test: It is recommended to test individual responsiveness to carglumic acid before initiating any long term treatment. 

① In a comatose child, start with a dose of 100 to 250 mg/kg/day and measure ammonia plasma concentration at least before each administration; it should normalise within a few hours after starting Carbaglu. 

② In a patient with moderate hyperammonaemia, administer a test dose of 100 to 200 mg/kg/day for 3 days with a constant protein intake and perform repeated determinations of ammonia plasma concentration (before and 1 hour after a meal); adjust the dose in order to maintain normal ammonia plasma levels. 

B. For isovaleric acidaemia, methylmalonic acidaemia and propionic acidaemia 

The treatment should start upon hyperammonaemia in organic acidaemia patients. The initial daily dose should be 100 mg/kg, up to 250 mg/kg if necessary. 

It should then be individually adjusted in order to maintain normal ammonia plasma levels. 

C. Renal impairment 

Dosage adjustment of this medicinal product is required according to Glomerular filtration rate (Glomerular filtration rate, GFR). 

①Patients with moderate renal impairment (GFR 30-59 mL/min) 

The recommended initial dose is 50 mg/kg/day to 125 mg/kg/day for patients presenting an hyperammonemia due to NAGS deficiency or organic acidaemia. In the long term use the daily dose will be in the range of 5 mg/kg/day to 50 mg/kg/day and should be adjusted individually in order to maintain normal ammonia plasma levels. 

②Patients with severe renal impairment (GFR ≤ 29 mL/min) 

The recommended initial dose is 15 mg/kg/day to 40 mg/kg/day for patients presenting an hyperammonaemia due to NAGS deficiency or organic acidaemia. In the long term use the daily dose will be in the range of 2 mg/kg/day to 20 mg/kg/day and should be adjusted individually in order to maintain normal ammonia plasma levels. 

2. Method of administration  

This medicine is for oral use ONLY (ingestion or via a nasogastric tube using a syringe, if necessary). 

It is recommended to divide the total daily dose into two to four doses to be given before meals or feedings. The breaking of the tablets in halves allows most of the required posology adjustments. Occasionally, the use of quarter tablets may also be useful to adjust the posology prescribed by the physician. 

The tablets must be dispersed in a minimum of 5-10 ml of water and ingested immediately or administered by fast push through a syringe via a nasogastric tube.