Drugs List

Therapeutic Indications

Uses

Hyperammonaemia due to N-acetylglutamate synthase deficiency
Hyperammonaemia due to organic acidaemias

Contraindications

Breastfeeding

Precautions and Warnings

Pregnancy

Not all available brands are licensed for all indications
Treatment to be initiated and supervised by a specialist
Monitor cardiac function
Monitor haematological parameters periodically
Monitor hepatic function
Monitor renal function

Protein restriction and arginine supplementation may be indicated in case of low protein tolerance.

Pregnancy and Lactation

Pregnancy

Carglumic acid should be used with caution during pregnancy.

There are no adequate data on the use of carglumic acid in pregnant women. Animal studies have revealed minimal developmental toxicity.

The use of all medication in pregnancy should be avoided whenever possible; particularly in the first trimester. Non-drug treatments should also be considered. When essential, a medication with the best safety record over time should be chosen, employing the lowest effective dose for the shortest possible time. Polypharmacy should be avoided. Teratogens taken in the pre-embryonic period, often quoted as lasting until 14 to 17 days post-conception, are believed to have an all-or-nothing effect. Where drugs have a short half-life, and when the date of conception is certain, this may allow women to be reassured where drug exposure has occurred within this time frame. Further advice may be available from the UK National Teratology Information Service (NTIS) and through ToxBase, available via password on the internet ( www.toxbase.org ) or if this is unavailable at the backup site ( www.toxbasebackup.org ).

Lactation

The manufacturer advises that carglumic acid is contraindicated during breastfeeding.

Although it is not known if carglumic acid is secreted in human breast milk, it has been shown to be present in the milk of lactating rats.

Neonates, infants born prematurely, those with low birth weight, those with an unstable gastrointestinal function or who have serious illnesses may require special consideration. For any infant, if a drug is prescribed to the nursing mother, it should be at the lowest practical dose and for the shortest time. When drug administration is unavoidable and breastfeeding is to continue, minimisation of exposure of the infant to the drug may sometimes be achieved by timing the maternal doses to just after a feeding episode. Infants exposed to drugs via breast milk should be monitored for unusual signs or symptoms. Interactions between the drug received by the infant from the mother's milk and medication prescribed for the infant should also be considered, for example, when the drug given to the infant may prevent metabolism of the drug received via breast milk.
Specialist advice is available from the UK Drugs in Lactation Advisory Service at https://www.midlandsmedicines.nhs.uk/content.asp?section=6&subsection=17&pageIdx=1

Side Effects

Bradycardia
Diarrhoea
Increase in serum transaminases
Increased sweating
Pyrexia
Rash
Vomiting

Presentation

Oral formulations of carglumic acid.

Drugs List

Therapeutic Indications

Uses

Hyperammonaemia due to N-acetylglutamate synthase deficiency
Hyperammonaemia due to organic acidaemias

Dosage

Adults

Children

Additional Dosage Information

Contraindications

Breastfeeding

Precautions and Warnings

Pregnancy

Not all available brands are licensed for all indications
Treatment to be initiated and supervised by a specialist
Monitor cardiac function
Monitor haematological parameters periodically
Monitor hepatic function
Monitor renal function

Protein restriction and arginine supplementation may be indicated in case of low protein tolerance.

Pregnancy and Lactation

Pregnancy

Carglumic acid should be used with caution during pregnancy.

There are no adequate data on the use of carglumic acid in pregnant women. Animal studies have revealed minimal developmental toxicity.

The use of all medication in pregnancy should be avoided whenever possible; particularly in the first trimester. Non-drug treatments should also be considered. When essential, a medication with the best safety record over time should be chosen, employing the lowest effective dose for the shortest possible time. Polypharmacy should be avoided. Teratogens taken in the pre-embryonic period, often quoted as lasting until 14 to 17 days post-conception, are believed to have an all-or-nothing effect. Where drugs have a short half-life, and when the date of conception is certain, this may allow women to be reassured where drug exposure has occurred within this time frame. Further advice may be available from the UK National Teratology Information Service (NTIS) and through ToxBase, available via password on the internet ( www.toxbase.org ) or if this is unavailable at the backup site ( www.toxbasebackup.org ).

Lactation

The manufacturer advises that carglumic acid is contraindicated during breastfeeding.

Although it is not known if carglumic acid is secreted in human breast milk, it has been shown to be present in the milk of lactating rats.

Neonates, infants born prematurely, those with low birth weight, those with an unstable gastrointestinal function or who have serious illnesses may require special consideration. For any infant, if a drug is prescribed to the nursing mother, it should be at the lowest practical dose and for the shortest time. When drug administration is unavoidable and breastfeeding is to continue, minimisation of exposure of the infant to the drug may sometimes be achieved by timing the maternal doses to just after a feeding episode. Infants exposed to drugs via breast milk should be monitored for unusual signs or symptoms. Interactions between the drug received by the infant from the mother's milk and medication prescribed for the infant should also be considered, for example, when the drug given to the infant may prevent metabolism of the drug received via breast milk.
Specialist advice is available from the UK Drugs in Lactation Advisory Service at https://www.midlandsmedicines.nhs.uk/content.asp?section=6&subsection=17&pageIdx=1

Side Effects

Bradycardia
Diarrhoea
Increase in serum transaminases
Increased sweating
Pyrexia
Rash
Vomiting

Overdosage

It is strongly recommended that the UK National Poisons Information Service be consulted on cases of suspected or actual overdose where there is doubt over the degree of risk or about appropriate management.

The following number will direct the caller to the relevant local centre (0844) 892 0111

Information may be obtained if you have access to ToxBase the primary clinical toxicology database of the National Poisons Information Service. This is available via password on the internet ( www.toxbase.org ) or if this is unavailable at the backup site ( www.toxbasebackup.org ).

Further Information

Last review date: February 2016

Reference Sources

Summary of Product Characteristics: Carbaglu 200mg dispersible tablets. Orphan Europe (UK) Ltd. Revised September 2017.

Summary of Product Characteristics: Ucedane 200mg dispersible tablets. Lucane Pharma. Revised June 2017.

NICE Evidence Services Available at: www.nice.org.uk Last accessed: 5 October 2017