Pharmacy Guide
Patient leaflets and SmPCs, drug interaction checker, official dosages and NHS pharmacy opening hours — all in one place.
Pharmacy Guide
Patient leaflets and SmPCs, drug interaction checker, official dosages and NHS pharmacy opening hours — all in one place.
Carglumic Acid 200 mg Dispersible Tablets
⚠ This medicine appears to have been discontinued
The electronic medicines compendium (emc) no longer publishes a Summary of Product Characteristics for this product, which usually means it is no longer marketed in the UK. The patient leaflet below is kept for reference, but the product may not be available.
If you were prescribed this medicine, other products containing Carglumic acid may still be available. Do not stop your treatment — ask your pharmacist or GP what to use instead.
What it is and what it is used for
for Your medicine is called Carglumic Acid 200 mg Dispersible Tablets. In this leaflet it will be called Carglumic Acid. Carglumic Acid can help to eliminate high ammonia levels in the blood. Ammonia is especially toxic for the brain and can lead, in severe cases, to reduced levels of consciousness and to coma. High levels of ammonia in the blood may be due to:
- the lack of a specific liver enzyme (N-acetylglutamate synthase). Patients with this rare disorder are not able to eliminate nitrogen waste, which builds up after eating protein. This disorder persists throughout the life of the affected patient, requiring lifelong treatment.
- isovaleric acidaemia, methylmalonic acidaemia or propionic acidaemia. Patients suffering from one of these disorders need treatment during the hyperammonaemia crisis.
What you need to know before you take it
e Carglumic Acid Do not take Carglumic Acid:
- if you are hypersensitive (allergic) to carglumic acid or any of the other ingredients of these tablets (see section 6 for a list of ingredients);
- during breast-feeding. Warnings and precautions Carglumic Acid treatment should be initiated under the supervision of a doctor experienced in treating metabolic disorders. Your doctor will evaluate your individual response to carglumic acid before starting any long term treatment. The dose should be individually adjusted in order to maintain normal ammonia plasma levels. Your doctor may prescribe a supplement called arginine, or restrict your protein intake.
In order to follow up your condition and your treatment, your doctor may examine your liver, kidneys, heart and blood on a regular basis. Other medicines and Carglumic Acid Please tell your doctor or pharmacist if you are taking, have recently taken or might take, any other medicines. Carglumic Acid with food and drink Take carglumic acid before meals or feeds. Disperse the tablets in at least 5 to 10 ml of water and take it immediately. The suspension will have a slightly acidic taste. Pregnancy and breast-feeding If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before taking this medicine. Pregnancy The effects of Carglumic Acid on pregnancy and the unborn child are not known. Please consult your doctor for advice if you are pregnant or planning to become pregnant. Breast-feeding Do not breast-feed your baby if you are taking Carglumic Acid. The excretion of carglumic acid into breast milk has not been studied in women. Nevertheless, carglumic acid has been shown to be present in the milk of lactating rats with potential toxic effects for their fed pups. Driving and using machines Effects on the ability to drive and use machines are not known. Carglumic Acid contains sodium This medicinal product contains up to 3 mg of sodium per tablet. The maximum recommended daily dose of this medicinal product contains 396 mg sodium (found in table salt). This is equivalent to 20% of the adult recommended maximum daily dietary intake for sodium. Talk to your doctor or pharmacist if you need 132 or more tablets daily for a prolonged period of time, especially if you have been advised to follow a low salt (sodium) diet.
How to take it
Carglumic Acid Always take Carglumic Acid exactly as your doctor has instructed you. You should check with your doctor or pharmacist if you are not sure. The usual dose: The initial daily dose is usually 100 mg per kilogram of body weight, up to a maximum of 250 mg per kilogram of body weight (for example, if you weigh 10 kg, you should take 1 g per day, or 5 tablets). For patients suffering from N-acetylglutamate synthase deficiency, in the long term, the daily dose usually ranges from 10 mg to 100 mg per kilogram of body weight. Your doctor will determine the dose suitable to you in order to maintain normal ammonia levels in your blood. Carglumic Acid should ONLY be given by mouth, or through a feeding tube into the stomach (using a syringe, if necessary).
When the patient is in hyperammonaemic coma, Carglumic Acid is administered by fast push through a syringe via the tube set up to feed them. Tell your doctor in case you are suffering from renal impairment. Your daily dose should be reduced. If you take more Carglumic Acid than you should: If you accidentally take too much of your medicine, tell your doctor at once or contact your nearest hospital casualty department immediately. Take your medicine and this leaflet with you. If you forget to take Carglumic Acid If you forget to take a dose take the next dose at the usual time. DO NOT take a double dose to make up for a forgotten dose. If you stop taking Carglumic Acid Do not stop taking Carglumic Acid without informing your doctor. If you have any further questions on the use of this product, ask your doctor or pharmacist.
Possible side effects
Like all medicines, Carglumic Acid can have side effects, although not everybody gets them. The following side effects were reported as follows:
- Common (may affect up to 1 in 10 people): increased sweating.
- Uncommon (may affect up to 1 in 100 people): reduced heart beat, diarrhoea, fever, vomiting, increased transaminases (enzymes that may indicate liver damage).
- Frequency not known: rash. If any of these side effects becomes serious, or if you notice any side effects not listed in this leaflet, please tell your doctor or pharmacist. Reporting of side effects If you get any side effects, talk to your doctor, pharmacist or nurse. This includes any possible side effects not listed in this leaflet. You can also report side effects directly via the Yellow Card Scheme at www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google Play or Apple App Store. By reporting side effects you can help provide more information on the safety of this medicine.
How to store it
Carglumic Acid Keep this medicine out of the sight and reach of children. Do not use this medicine after the expiry date which is stated on the tablet container, carton or blister after EXP. The expiry date refers to the last day of that month. Bottle: This medicinal product does not require any special storage conditions. After first opening of the tablet container: do not refrigerate, do not freeze. Discard 1 month after first opening.
Keep the container tightly closed in order to protect from moisture. Blister: This medicinal product does not require any special storage conditions. Do not throw away any medicines via wastewater or household waste. Ask your pharmacist how to throw away medicines you no longer use. These measures will help protect the environment.
Contents of the pack and other information
What Carglumic Acid contains The active substance is carglumic acid. Each tablet contains 200 mg of carglumic acid. The other ingredients are microcrystalline cellulose, croscarmellose sodium, sodium laurilsulfate, colloidal anhydrous silica, sodium stearyl fumarate. What Carglumic Acid looks like and contents of the pack Carglumic Acid 200 mg Dispersible Tablets are white to off-white elongated dispersible tablets, 18 mm x 6 mm, with three score marks on both side and engraved with 'N's on one side. The tablet can be divided into equal doses. Carglumic Acid is presented in containers or blisters of 5, 15 and 60 tablets. Not all pack sizes may be marketed. Marketing Authorisation Holder Waymade Medical, Sovereign House, Miles Gray Road, Basildon, Essex, SS14 3FR, United Kingdom Manufacturer Waymade Plc, Sovereign House, Miles Gray Road, Basildon, Essex, SS14 3FR, United Kingdom Or Waymade Plc, Josselin Road, Burnt Mills Industrial Estate, Basildon, Essex, SS13 1QF, United Kingdom Or Drehm Pharma GmbH, Grünbergstraße 15/3/3, 1120 Wien, Austria Or AcertiPharma B.V., Boschstraat 51, 4811 GC, Breda, Netherlands For any information about this medicine, please contact the local representative of the Marketing Authorisation Holder: Waymade Plc Sovereign House, Miles Gray Road, Basildon, Essex, SS14 3FR, United Kingdom This leaflet was last revised in April 2024. PL 06464/3072 Carglumic Acid 200 mg Dispersible Tablets
To request a copy of this leaflet in Braille, large print or audio format, contact the licence holder at the above address or telephone: 01268 535200 (select option Medical Information) / e-mail: [email protected]
Frequently asked questions about Carglumic Acid 200 mg Dispersible Tablets
How do I take Carglumic Acid 200 mg Dispersible Tablets?
Carglumic Acid 200 mg Dispersible Tablets comes as tablet containing 200mg. Always follow the dose your doctor or pharmacist has given you, and read the leaflet that comes with the medicine.
What is the active substance in Carglumic Acid 200 mg Dispersible Tablets?
The active substance in Carglumic Acid 200 mg Dispersible Tablets is carglumic acid.
Are there equivalent medicines to Carglumic Acid 200 mg Dispersible Tablets?
Medicines with the same active substance, strength and form include: Carbaglu 200 mg dispersible tablets, Ucedane 200 mg dispersible tablets. They are interchangeable only if your prescriber or pharmacist says so.
Where does this information come from?
This leaflet reproduces the patient information leaflet approved for Carglumic Acid 200 mg Dispersible Tablets, as published on the electronic medicines compendium (emc). The version printed inside your medicine’s packaging is the one that applies to you.
Can I get Carglumic Acid 200 mg Dispersible Tablets without a prescription?
Whether a medicine is available over the counter or on prescription only depends on its licence. Check the leaflet, or ask your pharmacist — they can tell you straight away.
About this leaflet
The text above reproduces the patient information leaflet approved for this medicine, restructured for easier reading.
- Source: electronic medicines compendium (emc), Datapharm
- Active substance: carglumic acid
- Official document: view the original leaflet on emc →
⚕For healthcare professionals — Summary of Product Characteristics (SmPC)Full SmPC: dosage, interactions, contraindications, warnings+
4.1. Therapeutic indications
Carglumic Acid 200 mg Dispersible Tablets are indicated in the treatment of:
• hyperammonaemia due to N-acetylglutamate synthase primary deficiency.
• hyperammonaemia due to isovaleric acidaemia.
• hyperammonaemia due to methylmalonic acidaemia.
• hyperammonaemia due to propionic acidaemia.
4.2. Posology and method of administration
Carglumic Acid 200 mg Dispersible Tablet treatment should be initiated under the supervision of a physician experienced in the treatment of metabolic disorders.
Posology
• For N-acetylglutamate synthase deficiency:
Based on clinical experience, 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 (see section 4.4).
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. As examples:
- 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 carglumic acid 200 mg dispersible tablets.
- 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.
• 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 (see section 4.4).
Renal impairment:
Caution is advised when administering Carglumic Acid to patients with impaired renal function.
Dosage adjustment is required according to GFR.
• Patients 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
• Patients 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
Paediatric population
The safety and effectiveness of Carglumic Acid for the treatment of pediatric patients (birth to 17 years of age) with acute or chronic hyperammonemia due to NAGS deficiency and acute hyperammonemia due to IVA, PA or MMA have been established, and based on these data, posology adjustments in neonates are not deemed necessary.
Method of administration:
This medicine is for oral use ONLY (ingestion or via a nasogastric tube using a syringe, if necessary).
Based on pharmacokinetic data and clinical experience, 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.
The suspension has a slightly acidic taste.
4.3. Contraindications
Hypersensitivity to the active substance or to any of the excipients listed in section 6.1.
Breast-feeding during the use of carglumic acid is contraindicated (see sections 4.6 and 5.3).
4.4. Special warnings and precautions for use
Therapeutic monitoring
Plasma levels of ammonia and amino acids should be maintained within normal limits.
As very few data on the safety of carglumic acid are available, systematic surveillance of liver, renal, cardiac functions and haematological parameters is recommended.
Nutritional management
Protein restriction and arginine supplementation may be indicated in case of low protein tolerance.
This medicinal product contains up to 3 mg sodium per tablet, equivalent to 0.15 % of the WHO recommended maximum daily intake for sodium.
The maximum daily dose of this product is equivalent to 20% of the WHO recommended maximum daily intake for sodium.
Carglumic Acid 200 mg Dispersible Tablets are considered high in sodium. This should be particularly taken into account for those on a low salt diet.
Use in patients with renal impairment
The dose of Carglumic Acid must be reduced in patients with renal impairment (see section 4.2)
4.5. Interaction with other medicinal products and other forms of interaction
No interaction studies have been performed.
4.6. Fertility, pregnancy and lactation
Pregnancy
For carglumic acid no clinical data on exposed pregnancies are available.
Animal studies have revealed minimal developmental toxicity (see section 5.3). Caution should be exercised when prescribing to pregnant women.
Breast-feeding
Although it is not known whether carglumic acid is secreted into human milk, it has been shown to be present in the milk of lactating rats (see section 5.3). Therefore, breast-feeding during the use of carglumic acid is contraindicated. (see section 4.3).
Fertility
No human data on the effect of carglumic acid on fertility are available. In rats, no adverse effects have been observed on male or female fertility with carglumic acid treatment (see section 5.3).
4.7. Effects on ability to drive and use machines
No studies on the effects on the ability to drive and use machines have been performed.
4.8. Undesirable effects
Reported adverse reactions are listed below, by system organ class and by frequency. Frequencies are defined as: very common (≥ 1/10), common (≥1/100 to <1/10) and uncommon (≥1/1,000 to <1/100).
Within each frequency grouping, undesirable effects are presented in order of decreasing seriousness.
- Undesirable effects in N-acetylglutamate synthase deficiency
Investigations
Uncommon: increased transaminases
Skin and subcutaneous tissue disorders
Common: increased sweating
Not known: rash
- Undesirable effects in organic acidaemia
Cardiac disorders
Uncommon: bradycardia
Gastrointestinal disorders
Uncommon: diarrhoea, vomiting
General disorders and Administration site conditions
Uncommon: pyrexia
Skin and subcutaneous tissue disorders
Not known: rash
Reporting of suspected adverse reactions
Reporting suspected adverse reactions after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals are asked to report any suspected adverse reactions via the Yellow Card Scheme at www.mhra.gov.uk/yellowcard or search for MHRA Yellow Card in the Google play or Apple App Store.
4.9. Overdose
In one patient treated with carglumic acid, where the dose was increased up to 750 mg/kg/day, symptoms of intoxication occurred which can be characterised as a sympathomimetic reaction: tachycardia, profuse sweating, increased bronchial secretion, increased body temperature and restlessness. These symptoms resolved once the dose was reduced.
💬 Ask about this leaflet
Ask anything about Carglumic Acid 200 mg Dispersible Tablets. The assistant answers only from this leaflet — if the leaflet does not cover it, it will say so. It does not give medical advice.
Answers come from the patient leaflet published on the electronic medicines compendium (emc). They are not medical advice. Ask a pharmacist or your GP if you are unsure. For urgent help call NHS 111, or 999 in an emergency.