Carbaglu

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ANNEX I 

 

SUMMARY OF PRODUCT CHARACTERISTICS 

 



 

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1. NAME OF THE MEDICINAL PRODUCT 

 

Carbaglu 200 mg dispersible tablets 

 

 

2. QUALITATIVE AND QUANTITATIVE COMPOSITION 

 

2.1 General description  

Each tablet contains 200 mg of carglumic acid. 

 

2.2 Qualitative and quantitative composition  

For a full list of excipients, see section 6.1 

 

 

3. PHARMACEUTICAL FORM 

 

Dispersible tablet 

The tablets are white and elongated with three score marks and engraved on one side. 

The tablet can be divided into equal halves. 

 

 

4. CLINICAL PARTICULARS 

 

4.1 Therapeutic indications 

 

Carbaglu is indicated in treatment of  

 hyperammonaemia due to N-acetylglutamate synthase primary deficiency. 

 hyperammonaemia due to isovaleric acidaemia. 

 hyperammonaemia due to methymalonic acidaemia. 

 hyperammonaemia due to propionic acidaemia. 

 

4.2 Posology and method of administration 

 

Carbaglu 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 Carbaglu. 

-  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. 



 

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 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). 

 

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. 

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. 

 

4.5 Interaction with other medicinal products and other forms of interaction 

 

No specific 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). 

 

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. 

 



 

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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), uncommon (≥1/1,000 to 1/100), rare 

(≥ 1/10,000 to < 1/1,000), very rare (< 1/10,000), not known (cannot be estimated from the available 

data). 

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 national reporting system 

listed in Appendix V. 

 

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. 

 

 

5. PHARMACOLOGICAL PROPERTIES 

 

5.1 Pharmacodynamic properties 

 

Pharmacotherapeutic group: Amino acids and derivatives; ATC code: A16AA05 

 

Mechanism of action 

Carglumic acid is a structural analogue of N-acetylglutamate, which is the naturally occurring 

activator of carbamoyl phosphate synthetase, the first enzyme of the urea cycle. 

Carglumic acid has been shown in vitro to activate liver carbamoyl phosphate synthetase. Despite a 

lower affinity of carbamoyl phosphate synthetase for carglumic acid than for N-acetylglutamate, 

carglumic acid has been shown in vivo to stimulate carbamoyl phosphate synthetase and to be much 



 

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more effective than N-acetylglutamate in protecting against ammonia intoxication in rats. This could 

be explained by the following observations: 

i) The mitochondrial membrane is more readily permeable for carglumic acid than for N-

acetylglutamate 

ii) Carglumic acid is more resistant than N-acetylglutamate to hydrolysis by aminoacylase present in 

the cytosol. 

 

Pharmacodynamic effects  

Other studies have been conducted in rats under different experimental conditions leading to 

increased ammonia availability (starvation, protein-free or high-protein diet). Carglumic acid was 

shown to decrease blood ammonia levels and increase urea levels in blood and urine, whereas the 

liver content of carbamoyl phosphate synthetase activators was significantly increased. 

 

Clinical efficacy and safety  

In patients with N-acetylglutamate synthase deficiency, carglumic acid was shown to induce a rapid 

normalisation of plasma ammonia levels, usually within 24 hours. When the treatment was instituted 

before any permanent brain damage, patients exhibited normal growth and psychomotor development. 

In patients with organic acidaemia (neonates and non-neonates), the treatment with carglumic acid 

induced a quick decrease of ammonia plasma levels, reducing the risk of neurological complications. 

 

5.2 Pharmacokinetic properties 

 

The pharmacokinetics of carglumic acid has been studied in healthy male volunteers using both 

radiolabelled and unlabelled product. 

 

Absorption 

After a single oral dose of 100 mg/kg body weight, approximately 30% of carglumic acid is estimated 

to be absorbed. At that dose-level, in 12 volunteers given Carbaglu tablets, plasma concentration 

peaked at 2.6 µg/ml (median; range 1.8-4.8) after 3 hours (median; range 2-4). 

 

Distribution 

The plasma elimination curve of carglumic acid is biphasic with a rapid phase over the first 12 hours 

after administration followed by a slow phase (terminal half life up to 28 hours). 

Diffusion into erythrocytes is non existent. Protein binding has not been determined. 

 

Metabolism 

A proportion of carglumic acid is metabolised. It is suggested that depending on its activity, the 

intestinal bacterial flora may contribute to the initiation of the degradation process, thus leading to a 

variable extent of metabolism of the molecule. One metabolite that has been identified in the faeces is 

glutamic acid. Metabolites are detectable in plasma with a peak at 36-48 hours and a very slow 

decline (half-life around 100 hours). 

The end product of carglumic acid metabolism is carbon dioxide, which is eliminated through the 

lungs. 

 

Elimination 

After a single oral dose of 100 mg/kg body weight, 9% of the dose is excreted unchanged in the urine 

and up to 60% in the faeces. 

 

Plasma levels of carglumic acid were measured in patients of all age categories, from newborn infants 

to adolescents, treated with various daily doses (7 – 122 mg/kg/day). Their range was consistent with 

those measured in healthy adults, even in newborn infants. Whatever the daily dose, they were slowly 

declining over 15 hours to levels around 100 ng/ml. 

 

 

 

 



 

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5.3 Preclinical safety data 

 

Safety pharmacology studies have shown that Carbaglu administered orally at doses of 250, 500, 

1000 mg/kg had no statistically significant effect on respiration, central nervous system and 

cardiovascular system. 

 

Carbaglu showed no significant mutagenic activity in a battery of genotoxicity tests performed in 

vitro (Ames test, human lymphocyte metaphase analysis) and in vivo (micronucleus test in rat). 

 

Single doses of carglumic acid up to 2800 mg/kg orally and 239 mg/kg intravenously did not induce 

any mortality or abnormal clinical signs in adult rats. In newborn rats receiving daily carglumic acid 

by oral gavage for 18 days as well as in young rats receiving daily carglumic acid for 26 weeks, the 

No Observed Effect Level (NOEL) was established at 500 mg/kg/day and the No Observed Adverse 

Effect Level (NOAEL) was established at 1000 mg/kg/day. 

 

No adverse effects have been observed on male or female fertility. In rats and rabbits no evidence has 

been seen of embryotoxicity, foetotoxicity or teratogenicity up to maternotoxic doses leading to fifty 

times exposure as compared to humans in rats and seven times in rabbits. Carglumic acid is secreted 

in the milk of lactating rats and although developmental parameters were unaffected, there were some 

effects on body weight / body weight gain of pups breast-fed by dams treated with 500 mg/kg/day and 

a higher mortality of pups from dams treated with 2000 mg/kg/day, a dose that caused 

maternotoxicity. The maternal systemic exposures after 500 and 2000 mg/kg/day were twenty five 

times and seventy times the expected human exposure. 

 

No carcinogenicity study has been conducted with carglumic acid. 

 

 

6. PHARMACEUTICAL PARTICULARS 

 

6.1 List of excipients 

 

Microcrystalline cellulose 

sodium laurilsulfate 

hypromellose 

croscarmellose sodium 

silica colloidal anhydrous 

sodium stearyl fumarate 

 

6.2 Incompatibilities 

 

Not applicable 

 

6.3 Shelf life 

 

36 months 

After first opening of the tablet container: 3 months 

 

6.4 Special precautions for storage 

 

Store in a refrigerator (2C – 8C) 

 

After first opening of the tablet container:  

Do not refrigerate. 

Do not store above 30C. 

Keep the container tightly closed in order to protect from moisture. 

 



 

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6.5 Nature and contents of container  

 

5-, 15- or 60- high density polyethylene tablet containers closed by a child resistant polypropylene cap 

with a desiccant unit. 

 

Not all pack sizes may be marketed.  

 

6.6 Special precautions for disposal  

 

No special requirements 

 

 

7. MARKETING AUTHORISATION HOLDER 

 

Recordati Rare Diseases 

Immeuble “Le Wilson” 

70, avenue du Général de Gaulle 

F-92800 Puteaux 

France 

 

 

8. MARKETING AUTHORISATION NUMBER(S) 

 

EU/1/02/246/001 (15 dispersible tablets) 

EU/1/02/246/002 (60 dispersible tablets) 

EU/1/02/246/003 (5 dispersible tablets) 

 

 

9. DATE OF FIRST AUTHORISATION/RENEWAL OF THE AUTHORISATION 

 

Date of first authorisation: 24 January 2003 

Date of renewal: 20 May 2008 

 

 

10. DATE OF REVISION OF THE TEXT 

 

 

 

Detailed information on this medicinal product is available on the website of the European Medicines 

Agency (EMA) http://www.ema.europa.eu/. 



 

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ANNEX II 

 

A. MANUFACTURERS RESPONSIBLE FOR BATCH 

RELEASE 

 

B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY 

AND USE 

 

C. OTHER CONDITIONS AND REQUIREMENTS OF THE  

MARKETING AUTHORISATION 

 

D.     CONDITIONS OR RESTRICTIONS WITH REGARD TO  

THE SAFE AND EFFECTIVE USE OF THE MEDICINAL 

PRODUCT 

 



 

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A. MANUFACTURERS RESPONSIBLE FOR BATCH RELEASE 

 

Name and address of the manufacturer responsible for batch release 

 

Recordati Rare Diseases 

Immeuble ‘Le Wilson’ 

70, avenue du Général de Gaulle 

F-92800 Puteaux 

France 

 

or 

 

Recordati Rare Diseases 

Eco River Parc 

30, rue des Peupliers 

F-92000 Nanterre 

France 

 

The printed package leaflet of the medicinal product must state the name and address of the 

manufacturer responsible for the release of the concerned batch. 

 

 

B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE 

 

Medicinal product subject to restricted medical prescription (See Annex I: Summary of Product 

Characteristics, section 4.2) 

 

 

C.  OTHER CONDITIONS AND REQUIREMENTS OF THE MARKETING 

AUTHORISATION 

 

 Periodic Safety Update Reports 
 

The requirements for submission of periodic safety update reports for this medicinal product 

are set out in the list of Union reference dates (EURD list) provided for under Article 107c(7) 

of Directive 2001/83/EC and any subsequent updates published on the European medicines 

web-portal. 

 

 

D. CONDITIONS OR RESTRICTIONS WITH REGARD TO THE SAFE AND 

EFFECTIVE USE OF THE MEDICINAL PRODUCT   

 

 Risk Management Plan (RMP) 

 

The MAH shall perform the required pharmacovigilance activities and interventions detailed in 

the agreed RMP presented in Module 1.8.2 of the Marketing Authorisation and any agreed 

subsequent updates of the RMP. 

 

An updated RMP should be submitted: 

 At the request of the European Medicines Agency; 

 Whenever the risk management system is modified, especially as the result of new information 

being received that may lead to a significant change to the benefit/risk profile or as the result of 

an important (pharmacovigilance or risk minimisation) milestone being reached. 

 



 

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ANNEX III 

 

LABELLING AND PACKAGE LEAFLET 



 

 11 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

A. LABELLING 



 

 12 

 

 

PARTICULARS TO APPEAR ON THE OUTER PACKAGING AND THE IMMEDIATE 

PACKAGING 

 

OUTER CARTON BOX AND TABLET CONTAINER LABEL X 5 TABLETS 

 

 

1. NAME OF THE MEDICINAL PRODUCT 

 

Carbaglu 200 mg dispersible tablets 

Carglumic acid  

 

 

2. STATEMENT OF ACTIVE SUBSTANCE(S) 

 

Each tablet contains 200 mg of carglumic acid. 

 

 

3. LIST OF EXCIPIENTS 

 

 

4. PHARMACEUTICAL FORM AND CONTENTS 

 

5 dispersible tablets 

 

 

5. METHOD AND ROUTE(S) OF ADMINISTRATION 

 

Oral use ONLY 

Read the package leaflet before use 

 

 

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 

OF THE REACH AND SIGHT OF CHILDREN 

 

Keep out of the reach and sight of children. 

 

 

7. OTHER SPECIAL WARNING(S), IF NECESSARY 

 

 

8. EXPIRY DATE 

 

EXP {MM/YYYY} 

Discard  3 months after first opening. 

Opened: 

9. SPECIAL STORAGE CONDITIONS 

 

Store in a refrigerator (2C – 8C) 

 

After first opening of the tablet container: do not refrigerate, do not store above 30C. 

Keep the container tightly closed in order to protect from moisture. 

 

 



 

 13 

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 

OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 

APPROPRIATE 

 

 

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER 

 

Recordati Rare Diseases 

Immeuble “Le Wilson” 

70, avenue du Général de Gaulle 

F-92800 Puteaux 

France 

 

 

12. MARKETING AUTHORISATION NUMBER(S) 

 

EU/1/02/246/003  

 

 

13. BATCH NUMBER 

 

Batch {number} 

 

 

14. GENERAL CLASSIFICATION FOR SUPPLY 

 

Medicinal product subject to medical prescription. 

 

 

15. INSTRUCTIONS ON USE 

 

 

16. INFORMATION IN BRAILLE 

 

Carbaglu 200 mg 

 

 

17. UNIQUE IDENTIFIER – 2D BARCODE 

 

2D barcode carrying the unique identifier included. 

 

 

18. UNIQUE IDENTIFIER - HUMAN READABLE DATA 

 

PC: 

SN:  

NN:  



 

 14 

 

PARTICULARS TO APPEAR ON THE OUTER PACKAGING AND THE IMMEDIATE 

PACKAGING 

 

OUTER CARTON BOX AND TABLET CONTAINER LABEL X 15 TABLETS 

 

 

1. NAME OF THE MEDICINAL PRODUCT 

 

Carbaglu 200 mg dispersible tablets 

Carglumic acid  

 

 

2. STATEMENT OF ACTIVE SUBSTANCE(S) 

 

Each tablet contains 200 mg of carglumic acid. 

 

 

3. LIST OF EXCIPIENTS 

 

 

4. PHARMACEUTICAL FORM AND CONTENTS 

 

15 dispersible tablets 

 

 

5. METHOD AND ROUTE(S) OF ADMINISTRATION 

 

Oral use ONLY 

Read the package leaflet before use 

 

 

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 

OF THE REACH AND SIGHT OF CHILDREN 

 

Keep out of the reach and sight of children. 

 

 

7. OTHER SPECIAL WARNING(S), IF NECESSARY 

 

 

8. EXPIRY DATE 

 

EXP {MM/YYYY} 

Discard 3 months after first opening. 

Opened: 

 

 

9. SPECIAL STORAGE CONDITIONS 

 

Store in a refrigerator (2C – 8C) 

 

After first opening of the tablet container: do not refrigerate, do not store above 30C. 

Keep the container tightly closed in order to protect from moisture. 

 



 

 15 

 

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 

OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 

APPROPRIATE 

 

 

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER 

 

Recordati Rare Diseases 

Immeuble “Le Wilson” 

70, avenue du Général de Gaulle 

F-92800 Puteaux 

France 

 

 

12. MARKETING AUTHORISATION NUMBER(S) 

 

EU/1/02/246/001 

 

 

13. BATCH NUMBER 

 

Batch {number} 

 

 

14. GENERAL CLASSIFICATION FOR SUPPLY 

 

Medicinal product subject to medical prescription. 

 

 

15. INSTRUCTIONS ON USE 

 

 

 

16. INFORMATION IN BRAILLE 

 

Carbaglu 200 mg 

 

 

17. UNIQUE IDENTIFIER – 2D BARCODE 

 

2D barcode carrying the unique identifier included. 

 

 

18. UNIQUE IDENTIFIER - HUMAN READABLE DATA 

 

PC:  

SN:  

NN:  

 



 

 16 

 

PARTICULARS TO APPEAR ON THE OUTER PACKAGING AND THE IMMEDIATE 

PACKAGING 

 

OUTER CARTON BOX AND TABLET CONTAINER LABEL X 60 TABLETS 

 

 

1. NAME OF THE MEDICINAL PRODUCT 

 

Carbaglu 200 mg dispersible tablets 

Carglumic acid  

 

 

2. STATEMENT OF ACTIVE SUBSTANCE(S) 

 

Each tablet contains 200 mg of carglumic acid. 

 

 

3. LIST OF EXCIPIENTS 

 

 

4. PHARMACEUTICAL FORM AND CONTENTS 

 

60 dispersible tablets 

 

 

5. METHOD AND ROUTE(S) OF ADMINISTRATION 

 

Oral use ONLY 

Read the package leaflet before use. 

 

 

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 

OF THE REACH AND SIGHT OF CHILDREN 

 

Keep out of the reach and sight of children. 

 

 

7. OTHER SPECIAL WARNING(S), IF NECESSARY 

 

 

8. EXPIRY DATE 

 

EXP {MM/YYYY} 

Discard 3 months after first opening. 

Opened: 

 

 

9. SPECIAL STORAGE CONDITIONS 

 

Store in a refrigerator (2C – 8C) 

 

After first opening of the tablet container: do not refrigerate, do not store above 30C. 

Keep the container tightly closed in order to protect from moisture. 

 



 

 17 

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 

OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 

APPROPRIATE 

 

 

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER 

 

Recordati Rare Diseases 

Immeuble “Le Wilson” 

70, avenue du Général de Gaulle 

F-92800 Puteaux 

France 

 

 

12. MARKETING AUTHORISATION NUMBER(S) 

 

EU/1/02/246/002 

 

 

13. BATCH NUMBER 

 

Batch {number} 

 

 

14. GENERAL CLASSIFICATION FOR SUPPLY 

 

Medicinal product subject to medical prescription. 

 

 

15. INSTRUCTIONS ON USE 

 

 

16. INFORMATION IN BRAILLE 

 

Carbaglu 200 mg 

 

 

17. UNIQUE IDENTIFIER – 2D BARCODE 

 

2D barcode carrying the unique identifier included. 

 

 

18. UNIQUE IDENTIFIER - HUMAN READABLE DATA 

 

PC:  

SN: 

NN:  

 



 

 18 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

B. PACKAGE LEAFLET 



 

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PACKAGE LEAFLET: INFORMATION FOR THE USER 

 

Carbaglu 200 mg dispesible tablets 

Carglumic acid 

 

Read all of this leaflet carefully before you start taking this medicine. 

- Keep this leaflet. You may need to read it again. 

- If you have further questions, please ask your doctor or your pharmacist. 

- This medicine has been prescribed for you personally and you should not pass it on to others. It 

may harm them, even if their symptoms are the same as yours. 

- If any of the side effects gets serious, or if you notice any side effects not listed in this leaflet, 

please tell your doctor or pharmacist. 

 

In this leaflet:  

1. What Carbaglu is and what it is used for 

2. Before you take Carbaglu 

3. How to take Carbaglu 

4. Possible side effects 

5 How to store Carbaglu 

6. Further information 

 

 

1. WHAT CARBAGLU IS AND WHAT IT IS USED FOR 

 

Carbaglu can help eliminating excessive ammonia plasma levels (elevated ammonia level in the 

blood). Ammonia is especially toxic for the brain and leads, in severe cases, to reduced levels of 

consciousness and to coma. 

Hyperammonaemia 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 during the entire life of the affected patient and therefore the need for this 

 treatment is lifelong. 

 isovaleric acidaemia, methylmalonic acidaemia or propionic acidaemia. Patients suffering from 

one of these disorders need treatment during the hyperammonaemia crisis. 

 

 

2. BEFORE YOU TAKE CARBAGLU 

 

Do not take Carbaglu: 

Do not take Carbaglu if you are hypersensitive (allergic) to carglumic acid or any of the other 

ingredients of Carbaglu. 

Do not take Carbaglu during breast-feeding 

 

Take special care with Carbaglu: 

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

treatment of metabolic disorders. 

 

Your doctor will evaluate your individual responsiveness to carglumic acid before initiating any long 

term treatment.  

The dose should be individually adjusted in order to maintain normal ammonia plasma levels. 

 

Your doctor may prescribe supplemental arginine or restrict your protein intake. 

 

In order to follow-up your condition and your treatment, your doctor may examine your liver, your 

kidneys, your heart and your blood on a regular basis. 

 



 

 20 

Taking other medicines 

Please tell your doctor or pharmacist if you are taking or have recently taken any other medicines, 

including medicines obtained without a prescription. 

 

Taking Carbaglu with food and drink 

Carbaglu must be taken orally before meals or feedings. 

The tablets must be dispersed in a minimum of 5 to 10 ml of water and taken immediately. The 

suspension has a slightly acidic taste. 

 

Pregnancy and Breast-feeding 

The effects of Carbaglu on pregnancy and the unborn child are not known. Please consult your doctor 

for advice if you are pregnant or planning to become pregnant. 

The excretion of carglumic acid into breast milk has not been studied in women. Nevertheless, as 

carglumic acid has been shown to be present in the milk of lactating rats with potential toxic effects 

for their fed pups, you should not breast feed your baby if you are taking Carbaglu. 

 

Driving and using machines 

Effects on the ability to drive and use machines are not known. 

 

 

3. HOW TO TAKE CARBAGLU 

 

Always take Carbaglu exactly as your doctor has instructed you. You should check with your doctor 

or pharmacist if you are unsure. 

 

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 weight 10kg, you should take 1g 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. 

 

Carbaglu should ONLY be administered by mouth or via a feeding tube into the stomach (using a 

syringe, if necessary). 

 

When the patient is in hyperammonaemic coma, Carbaglu is administered by fast push through a 

syringe via the tube set up and used to feed you. 

 

If you take more Carbaglu than you should 

Ask your doctor or pharmacist for advice. 

 

If you forget to take Carbaglu 

Do not take a double dose to make up for forgotten individual doses. 

 

If you stop taking Carbaglu 

Do not stop Carbaglu without informing your doctor. 

 

If you have any further questions on the use of this product, ask your doctor or pharmacist. 

 

 

 

 

 

 



 

 21 

4. POSSIBLE SIDE EFFECTS 

 

Like all medicines, Carbaglu can have side effects, although not everybody gets them. 

 

The following side effects were reported as follows: very common (occurring in at least one in 10 

patients), common (occurring in at least one in 100 patients), uncommon (occurring in at least one 

in1,000 patients), rare (occurring in at least one in 10,000 patients), very rare (occurring in at least one 

in 100,000 patients) and not known (frequency cannot be estimated from the available data). 

 Common: increased sweating 

 Uncommon: bradycardia (decreased frequency of the heart), diarrhoea, fever, increased 

transaminases, vomiting 

 Not known: rash 

 

If any of the side effects gets 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 or pharmacist. This includes any possible side effects 

not listed in this leaflet. You can also report side effects directly via the national reporting system 

listed in Appendix V. By reporting side effects you can help provide more information on the safety of 

this medicine. 

 

 

5. HOW TO STORE CARBAGLU 

 

Keep out of the reach and sight of children. 

 

Do not use after the expiry date stated on the tablet container. 

 

Store in a refrigerator (2C – 8C). 

 

After first opening of the container: do not refrigerate, do not store above 30C. 

Keep the container tightly closed in order to protect from moisture. 

Write the date of opening on the tablet container. Discard 3 months after first opening. 

 

 

6. FURTHER INFORMATION 

 

What Carbaglu contains  

- The active substance is carglumic acid. Each tablet contains 200 mg of carglumic acid. 

- The other ingredients are microcrystalline cellulose, sodium laurilsulfate, hypromellose, 

croscarmellose sodium, silica colloidal anhydrous, sodium stearyl fumarate. 

 

What Carbaglu looks like and contents of the pack 

Carbaglu 200mg tablet is a bar-shaped tablet, with 4 punches on one side with 3 break-mark sides. 

Carbaglu is presented in a plastic container of 5, 15 and 60 tablets which is closed with a child 

resistant cap.  

 

Marketing Authorisation Holder 

Recordati Rare Diseases 

Immeuble “Le Wilson” 

70, avenue du Général de Gaulle 

F-92800 Puteaux 

France 

Tel: + 33 1 4773 6458 

Fax: + 33 1 4900 1800 



 

 22 

 

Manufacturer 

Recordati Rare Diseases 

Immeuble “Le Wilson” 

70, Avenue du Général de Gaulle 

F-92800 Puteaux 

France 

 

or 

 

Recordati Rare Diseases 

Eco River Parc 

30, rue des Peupliers 

F-92000 Nanterre 

France 

 

For any information about this medicinal product, please contact the local representative of the 

Marketing Authorisation Holder. 

 

Belgique/België/Belgien 

Recordati 

Tél/Tel: +32 2 46101 36 

Lietuva 

Recordati AB. 

Tel: + 46 8 545 80 230 

Švedija 

 

България 

Recordati Rare Diseases 

Teл.: +33 (0)1 47 73 64 58 

Франция  

 

Luxembourg/Luxemburg 

Recordati 

Tél/Tel: +32 2 46101 36 

Belgique/Belgien 

 

Česká republika 

Recordati Rare Diseases 

Tel: +33 (0)1 47 73 64 58 

Francie 

 

Magyarország 

Recordati Rare Diseases 

Tel: +33 (0)1 47 73 64 58 

Franciaország  

  

Danmark 

Recordati AB. 

Tlf : +46 8 545 80 230 

Sverige 

 

Malta 

Recordati Rare Diseases 

Tel: +33 1 47 73 64 58 

Franza  

 

Deutschland 

Recordati Rare Diseases Germany GmbH 

Tel: +49 731 140 554 0 

Nederland 

Recordati 

Tel: +32 2 46101 36 

België 

 

Eesti 

Recordati AB. 

Tel: + 46 8 545 80 230 

Rootsi 

 

Norge 

Recordati AB. 

Tlf : +46 8 545 80 230 

Sverige  

 

Ελλάδα 

Recordati Hellas 

Τηλ: +30 210 6773822 

 

Österreich 

Recordati Rare Diseases Germany GmbH 

Tel: +49 731 140 554 0 

Deutschland 

 

 

 



 

 23 

España 

Recordati Rare Diseases Spain S.L.U. 

Tel: + 34 91 659 28 90 

Polska 

Recordati Rare Diseases 

Tel: +33 (0)1 47 73 64 58 

Francja  

 

France 

Recordati Rare Diseases 

Tél: +33 (0)1 47 73 64 58 

 

Portugal 

Jaba Recordati S.A. 

Tel: +351 21 432 95 00 

 

Hrvatska 

Recordati Rare Diseases 

Tél: +33 (0)1 47 73 64 58 

Francuska 

 

România 

Recordati Rare Diseases 

Tel: +33 (0)1 47 73 64 58 

Franţa  

 

Ireland 

Recordati Rare Diseases 

Tel: +33 (0)1 47 73 64 58 

France 

Slovenija  

Recordati Rare Diseases 

Tel: +33 (0)1 47 73 64 58 

Francija  

  

Ísland 

Recordati AB. 

Simi:+46 8 545 80 230 

Svíþjóð 

 

Slovenská republika 

Recordati Rare Diseases 

Tel: +33 (0)1 47 73 64 58 

Francúzsko  

 

Italia 

Recordati Rare Diseases Italy Srl 

Tel: +39 02 487 87 173 

 

Suomi/Finland 

Recordati AB. 

Puh/Tel : +46 8 545 80 230 

Sverige 

 

Κύπρος 

Recordati Rare Diseases 

Τηλ : +33 1 47 73 64 58 
Γαλλία 

 

Sverige 

Recordati AB. 

Tel : +46 8 545 80 230 

Latvija 

Recordati AB. 

Tel: + 46 8 545 80 230 

Zviedrija 

 

United Kingdom 

Recordati Rare Diseases UK Ltd. 

Tel: +44 (0)1491 414333 

 

 

 

This leaflet was last approved on 

 

 

Detailed information on this medicine is available on the European Medicines Agency web site: 

http://www.ema.europa.eu. There are also links to other websites about rare diseases and treatments. 

 


	SUMMARY OF PRODUCT CHARACTERISTICS
	A. MANUFACTURERS RESPONSIBLE FOR BATCH RELEASE
	B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE
	C. OTHER CONDITIONS AND REQUIREMENTS OF THE MARKETING AUTHORISATION
	D. CONDITIONS OR RESTRICTIONS WITH REGARD TO THE SAFE AND EFFECTIVE USE OF THE MEDICINAL PRODUCT
	A. LABELLING
	B. PACKAGE LEAFLET