Cystadane

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ANNEX I 
 

SUMMARY OF PRODUCT CHARACTERISTICS 
 



 

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1. NAME OF THE MEDICINAL PRODUCT 
 

Cystadane 1 g oral powder  

 

 

2. QUALITATIVE AND QUANTITATIVE COMPOSITION 
 

1 g of powder contains 1 g of betaine anhydrous.  

 

For the full list of excipients, see section 6.1. 

 

 

3. PHARMACEUTICAL FORM 
 

Oral powder. 

White crystalline free flowing powder. 

 

 

4. CLINICAL PARTICULARS 
 

4.1 Therapeutic indications 
 

Adjunctive treatment of homocystinuria, involving deficiencies or defects in: 

 cystathionine beta-synthase (CBS), 

 5,10-methylene-tetrahydrofolate reductase (MTHFR), 

 cobalamin cofactor metabolism (cbl). 
 

Cystadane should be used as supplement to other therapies such as vitamin B6 (pyridoxine), vitamin 

B12 (cobalamin), folate and a specific diet. 

 

4.2 Posology and method of administration 
 

Cystadane treatment should be supervised by a physician experienced in the treatment of patients with 

homocystinuria. 

 

Posology 

Children and Adult 

The recommended total daily dose is 100 mg/kg/day given in 2 doses daily. However, the dose should 

be individually titrated according to plasma levels of homocysteine and methionine. In some patients 

doses above 200 mg/ kg/day were needed to reach therapeutic goals. Caution should be exercised with 

up-titrating doses for patients with CBS deficiency due to the risk for hypermethioninaemia. 

Methionine levels should be closely monitored in these patients.  

 

Special populations 

Hepatic or renal impairment 

Experience with betaine anhydrous therapy in patients with renal insufficiency or non-alcoholic 

hepatic steatosis has demonstrated no need to adapt the dose regimen of Cystadane.  

 

Method of administration 

The bottle should be lightly shaken before opening. Three measuring spoons are provided which 

dispense either 100 mg, 150 mg or 1 g of betaine anhydrous. It is recommended that a heaped 

measuring spoon is removed from the bottle and a flat surface e.g. base of a knife is drawn across the 

top of the measure. This will give the following doses: small measure 100 mg, middle size 

measure  150 mg and large measure 1 g of betaine anhydrous. 



 

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The powder should be mixed with water, juice, milk, formula or food until completely dissolved and 

ingested immediately after mixing. 
 

Therapeutic monitoring  

The aim of treatment is to keep plasma levels of total homocysteine below 15 µM or as low as 

possible. The steady-state response usually occurs within a month. 

 

4.3 Contraindications 
 

Hypersensitivity to the active substance. 

 

4.4 Special warnings and precautions for use 
 

Uncommon cases of severe cerebral oedema associated with hypermethioninemia were reported with 

betaine anhydrous therapy in patients with CBS deficiency (see section 4.8). Complete recovery was 

seen after treatment discontinuation: 

- The plasma methionine concentrations should be kept below 1000 µM. It is recommended to 
measure plasma methionine level at start of treatment and about annually or biannually 

thereafter. If methionine increases particularly above the first safety threshold of 700 µmol/L, 

patient should be monitored more frequently and compliance with diet should be checked. In 

order to reduce methionine levels, modification of diet as well as dose reduction of Cystadane or 

temporal interruption of Cystadane treatment should be considered. 

- If any symptoms of cerebral oedema like morning headaches with vomiting and/or visual changes 
appear, plasma methionine level and compliance to the diet should be checked and treatment with 

Cystadane interrupted.  

- If symptoms of cerebral oedema recur after re-introduction of treatment then betaine anhydrous 
therapy should be discontinued indefinitely. 

 

To minimise the risk of potential drug interactions, it is advisable to leave 30 minutes between the 

intake of betaine anhydrous and amino acids mixtures and/or medicinal products containing vigabatrin 

and GABA analogues (see section 4.5).  

 

4.5 Interaction with other medicinal products and other forms of interaction 
 

No interaction studies have been performed.  

Based on in vitro data, betaine anhydrous might interact with amino acids mixtures and medicinal 

products containing vigabatrin and GABA analogues. 

 

4.6 Fertility, pregnancy and lactation 

 

Pregnancy 

Data on a limited number of exposed pregnancies indicate no adverse event of betaine anhydrous on 

pregnancy or on the health of the foetus/newborn child. To date, no other relevant epidemiologic data 

are available. Animal reproduction studies have not been conducted. During pregnancy, administering 

betaine anhydrous in addition to pyridoxine, folate, anticoagulant and diet under close monitoring of 

plasma homocysteine would be compatible with good maternal and foetal outcomes. However, 

Cystadane should not be used during pregnancy unless clearly necessary. 

  

Breast-feeding 
It is not known whether betaine anhydrous is excreted in human milk (although its metabolic 

precursor, choline, occurs at high levels in human milk).  Because of lack of data, caution should be 

exercised when prescribing Cystadane to breast-feeding women.  

 

Fertility 

No data is available. 



 

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4.7 Effects on ability to drive and use machines 
 

Cystadane has no or negligible influence on the ability to drive and use machines. 

 

4.8 Undesirable effects 

 

Summary of the safety profile 
In general, adverse reactions seen with betaine anhydrous therapy appeared to be not serious and are 

mainly related to the gastrointestinal system. Gastrointestinal disorders like diarrhoea, glossitis, nausea, 

stomach discomfort, vomiting and dental disorders may occur uncommonly. 

The most commonly reported adverse reaction during treatment is blood methionine increased. 

Complete recovery was seen after treatment discontinuation (see section 4.4).  

 

Tabulated list of adverse reactions 

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). Within each frequency 

grouping, adverse reactions are presented in order of decreasing seriousness. 

 

Metabolism and nutrition disorders Uncommon: anorexia 

Psychiatric disorders Uncommon: agitation, irritability 

Nervous system disorders Uncommon: brain oedema* 

Gastrointestinal disorders 

 

Uncommon: diarrhoea, glossitis, nausea, stomach 

discomfort, vomiting  

Skin and subcutaneous tissue disorders Uncommon: hair loss, hives, skin odour abnormal 

Renal and urinary disorders Uncommon: urinary incontinence 

Investigations Very common: blood methionine increased* 

 

Description of selected adverse reactions 

*Uncommon cases of severe cerebral oedema and hypermethioninemia were reported within 2 weeks 

to 6 months of starting betaine anhydrous therapy in patients with CBS deficiency, with complete 

recovery after treatment discontinuation.  

Symptoms of cerebral oedema include morning headaches with vomiting and/or visual changes  

High increases in plasma methionine levels in a range from 1,000 to 3,000 µM were noted in these 

patients. As cerebral oedema has also been reported in patients with hypermethioninemia, secondary 

hypermethioninemia due to betaine anhydrous therapy has been postulated as a possible mechanism of 

action. 

For specific recommendations, see section 4.4. 

 

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 
 

No case of overdose has been reported. 

 

 



 

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5. PHARMACOLOGICAL PROPERTIES 

 

5.1  Pharmacodynamic properties 

 
Pharmacotherapeutic group: Other alimentary tract and metabolism products, ATC code: A16AA06. 

 

Mechanism of action 

Betaine anhydrous was shown to lower plasma homocysteine levels in the three types of 

homocystinuria, i.e. CBS deficiency; MTHFR deficiency and cbl defect. The extent of this effect was 

dependent on the absolute degree of hyperhomocysteinemia, being higher in severe 

hyperhomocysteinemia. 
 

Pharmacodynamic effects 

Betaine anhydrous acts as a methyl group donor in the remethylation of homocysteine to methionine 

in patients with homocystinuria. As a result, plasma levels of homocysteine should decrease in these 

patients, to 20-30 % of pre-treatment levels. 

Betaine anhydrous has also been shown to increase plasma methionine and S-adenosyl methionine 

(SAM) levels in patients with MTHFR deficiency and cbl defects. In CBS-deficient patients without 

dietary restriction of methionine, excessive accumulation of methionine has been observed. 

Betaine anhydrous supplementation was shown to improve the metabolic abnormalities in the 

cerebrospinal fluid of patients with homocystinuria. 

 

Clinical efficacy and safety 
Elevated homocysteine plasma levels are associated with cardiovascular events (such as thrombosis), 

osteoporosis, skeletal abnormalities, and optic lens dislocation. In observational studies, clinical 

improvement (cardiovascular and neurodevelopmental) was reported by the treating physician in about 

75% of patients taking betaine anhydrous. Most of these patients were also receiving other treatments 

such as vitamin B6 (pyridoxine), vitamin B12 (cobalamin) and folate with variable biochemical 

responses. In most cases, adding betaine anhydrous resulted in a further reduction in plasma 

homocysteine level. It is likely that due to the multiple nature of therapy (dietary, pharmaceutical, 

supportive) in these patients, there may be an element of overestimation in the clinical effects of 

betaine anhydrous treatment. Late detection of homocystinuria in symptomatic state is responsible for 

residual morbidity due to irreversible damage to connective tissue (ophtalmological, skeletal) that 

cannot be corrected by further therapy. The available clinical data do not allow correlating posology 

and clinical efficacy. There is no evidence of development of tolerance. 

 

In a few cases, increased plasma methionine levels were associated with cerebral oedema (see sections 

4.4 and 4.8). 
 

Monitoring plasma homocysteine levels has demonstrated that the onset of action of betaine 

anhydrous occurred within several days and that a steady-state response was achieved within one 

month.   

 

Paediatric population 

In paediatric patients less than 10 years of age, the usual effective dose regimen is  100 mg/kg/day 

given in 2 doses daily; increasing the frequency above twice daily and/or the dose 

above  150 mg/kg/day does not improve the homocysteine-lowering effect.   

 

Monitoring betaine plasma concentrations does not help to define the efficacy of treatment, since these 

concentrations do not directly correspond to the flux through the cytosolic betaine homocysteine 

methyl transferase pathway. 

 

 

 



 

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5.2 Pharmacokinetic properties  
 

The pharmacokinetic data of homocystinuric patients on long-term betaine anhydrous supplementation 

are very similar to those of healthy volunteers. This demonstrates that differences in betaine anhydrous 

kinetics are most probably due to betaine anhydrous depletion in untreated homocystinuria and are 

only meaningful for the initial treatment. 

 
Absorption 

The absolute bioavailability of betaine anhydrous has not been determined. In healthy adult volunteers 
(age between 21 to 49 years), after a single oral dose of betaine anhydrous (50 mg/kg), absorption 

was rapid (tmax = 0.9 ± 0.3 hours and a Cmax = 0.9 ± 0.2 mM).  
After a repeated dose regimen of 100 mg/kg/day for 5 days, the absorption kinetics did not change. 

 

Distribution 

Betaine anhydrous was rapidly distributed into a relatively large volume (V/F = 1.3 l/kg). 
After a repeated dose regiment of 100 mg/kg/day for 5 days, the distribution half life was prolonged 

significantly (up to 36 h), indicating saturable transport and redistribution processes. 

 

Biotransformation 

Betaine anhydrous is a methyl group donor 
 

Elimination 

With a slow elimination rate (mean half life = 14 h, mean total body clearance, CL/F, = 84 ml/h/kg), 
renal clearance is negligible (5% of total body clearance), assuming 100% bioavailability.  

 

5.3 Preclinical safety data 

 

At high doses, a CNS depressant effect and irritation of the gastrointestinal tract was seen in rats. 

Long-term carcinogenicity and reproductive toxicity studies have not been conducted on betaine 

anhydrous. A standard battery of genotoxicity test reveals no specific hazard for humans. 

 

 

6. PHARMACEUTICAL PARTICULARS 

 

6.1 List of excipients 
 

None. 
 

6.2 Incompatibilities 
 

Not applicable. 

 

6.3 Shelf life 
 

Unopened bottle: 3 years  

After the first opening: 3 months. 

 

6.4 Special precautions for storage 

 

Do not store above 25C. 

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

For storage conditions after first opening of the medicinal product, see section 6.3. 

 

 



 

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6.5 Nature and contents of container 
 

HDPE bottles with a child resistant closure. 

Each pack contains 1 bottle with 180 g of powder and three measuring spoons. 
 

 

6.6 Special precautions for disposal 
 

Any unused medicinal product or waste material should be disposed of in accordance with local 

requirements. 

 

 

7. MARKETING AUTHORISATION HOLDER 
 

Recordati Rare Diseases 

Immeuble “Le Wilson” 

70, Avenue du General de Gaulle  

F-92 800 Puteaux 

France 

 

 

8. MARKETING AUTHORISATION NUMBER(S)  

 

EU/1/06/379/001 

 

 

9. DATE OF FIRST AUTHORISATION/RENEWAL OF THE AUTHORISATION 
 

Date of first authorisation: 15 February 2007 

Date of latest renewal: 21 November 2016 

 

 

10. DATE OF REVISION OF THE TEXT 

 

 

 

 

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

Agency http://www.ema.europa.eu. 

 

  



 

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 ANNEX II 

 

A. MANUFACTURER 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 

 

 

 

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 

  



 

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A. LABELLING 
  



 

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PARTICULARS TO APPEAR ON THE OUTER PACKAGING  

 

OUTER CARTON 

 

 

1. NAME OF THE MEDICINAL PRODUCT 

 

Cystadane 1 g oral powder  

Betaine anhydrous 

 

 

2. STATEMENT OF ACTIVE SUBSTANCE(S) 

 

1 g of powder contains 1 g of betaine anhydrous. 

 

 

3. LIST OF EXCIPIENTS 

 

 

4. PHARMACEUTICAL FORM AND CONTENTS 

 

180 g of oral powder and three measuring spoons. 

Three measuring spoons (green, blue, pink) dispense 100mg, 150mg or 1g of betaine anhydrous. 

 

 

5. METHOD AND ROUTE(S) OF ADMINISTRATION 

 

Lightly shake the bottle before opening. 
Read the package leaflet before use. 

Oral use. 

 

 

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

OF THE SIGHT AND REACH OF CHILDREN 

 

Keep out of the sight and reach of children. 

 

 

7. OTHER SPECIAL WARNING(S), IF NECESSARY 

 

 

8. EXPIRY DATE 

 

EXP 
Shelf life after the first opening: 3 months. 

 

 

9. SPECIAL STORAGE CONDITIONS 

 

Do not store above 25C. 

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



 

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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 General de Gaulle  

F-92 800 Puteaux 
France 

 

 

12. MARKETING AUTHORISATION NUMBER(S) 

 

EU/1/06/379/001  

 

 

13. BATCH NUMBER 

 

Batch 

 

14. GENERAL CLASSIFICATION FOR SUPPLY 

 

Medicinal product subject to medical prescription. 

 

 

15. INSTRUCTIONS ON USE 

 

 

16. INFORMATION IN BRAILLE 

 

Cystadane 1 g oral powder 

 

 

17. UNIQUE IDENTIFIER – 2D BARCODE 

 

2D barcode carrying the unique identifier included. 

 

 

18. UNIQUE IDENTIFIER - HUMAN READABLE DATA 

 

PC:  

SN:  
NN:   



 

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PARTICULARS TO APPEAR ON THE IMMEDIATE PACKAGING 

 

BOTTLE LABEL 

 

 

1. NAME OF THE MEDICINAL PRODUCT 

 

Cystadane 1 g oral powder  

Betaine anhydrous 

 

 

2. STATEMENT OF ACTIVE SUBSTANCE(S) 

 

1 g of powder contains 1 g of betaine anhydrous. 

Three measuring spoons (green, blue, pink) dispense 100mg, 150mg or 1g of betaine anhydrous. 

 

 

3. LIST OF EXCIPIENTS 

 

 

4. PHARMACEUTICAL FORM AND CONTENTS 

 

180 g of oral powder. 

 

 

5. METHOD AND ROUTE(S) OF ADMINISTRATION 

 

Lightly shake the bottle before opening. 
Read the package leaflet before use 

Oral use. 
 

 

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

OF THE SIGHT AND REACH OF CHILDREN 

 

Keep out of the sight and reach of children. 

 

 

7. OTHER SPECIAL WARNING(S), IF NECESSARY 

 

 

8. EXPIRY DATE 

 

EXP 
Shelf life after the first opening: 3 months. 

Opened: 
 

 

9. SPECIAL STORAGE CONDITIONS 

 

Do not store above 25C. 

Keep the bottle 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 General de Gaulle  

F-92 800 Puteaux 
France 

 

 

12. MARKETING AUTHORISATION NUMBER(S) 

 

EU/1/06/379/001  

 

 

13. BATCH NUMBER 

 

Batch 

 

 

14. GENERAL CLASSIFICATION FOR SUPPLY 

 

Medicinal product subject to medical prescription. 

 

 

15. INSTRUCTIONS ON USE 

 

 

16. INFORMATION IN BRAILLE 

 

Cystadane 1 g oral powder 

 

 

17. UNIQUE IDENTIFIER – 2D BARCODE 

 

2D barcode carrying the unique identifier included. 

 

 

18. UNIQUE IDENTIFIER - HUMAN READABLE DATA 

 

PC: 

SN:  

NN:   



 

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B. PACKAGE LEAFLET 



 

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Package leaflet: Information for the patient 

 

Cystadane 1 g oral powder 

Betaine anhydrous 

 

Read all of this leaflet carefully before you start taking this medicine because it contains 

important information for you. 
- Keep this leaflet. You may need to read it again. 
- If you have any further questions, ask your doctor or your pharmacist. 
- This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, 

even if their signs of illness are the same as yours. 

- If you get any side effects, talk to your doctor or pharmacist. This includes any possible side 
effects not listed in this leaflet. See section 4. 

 

What is in this leaflet 
1. What Cystadane is and what it is used for 

2. What do you need to know before you take Cystadane 

3. How to take Cystadane 

4. Possible side effects 

5 How to store Cystadane 

6. Contents of the pack and other information 

 

 

1. What Cystadane is and what it is used for 
 

Cystadane contains betaine anhydrous which is intended to be an adjunctive treatment of 

homocystinuria, an inherited (genetic) disease where the amino acid methionine cannot be broken 

down completely by the body. 

 

Methionine is present in regular food protein (e.g. meat, fish, milk, cheese, eggs). It is converted into 

homocysteine which is then normally converted into cysteine during digestion. Homocystinuria is a 

disease caused by the accumulation of homocysteine which is not converted to cysteine and is 

characterised by formation of clots in the veins, bone weakness, and skeletal and crystalline lens 

abnormalities. The use of Cystadane together with other treatments such as vitamin B6, vitamin B12, 

folate and a specific diet aims to reduce the elevated homocysteine levels in your body.  

 

 

2. What you need to know before you take Cystadane 
 

Do not take Cystadane 
If you are allergic to betaine anhydrous. 

 

Warnings and precautions  

Talk to your doctor or pharmacist before taking Cystadane. 

If you notice side effects like headaches, vomiting or a change in your vision and you are of the 

homocystinuria subtype called CBS (cystathionine beta-synthase deficiency), please contact your 

doctor immediately, they could be signs of a swelling in the brain (cerebral oedema). In that case your 

doctor will monitor your methionine level in your body and may review your diet. Your treatment 

with Cystadane may need to be interrupted. 

If you are treated with Cystadane and with an amino-acid mixture and if you need to take other 

medicines at the same time, leave 30 minutes between the intake (see “section “Other medicines and 

Cystadane”). 



 

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Other medicines and Cystadane 
Tell your doctor if you are taking, have recently taken or might take any other medicines. 

If you are taking amino-acid mixture or medicines such as vigabatrin or Gaba analogues (medicine 

used to treat epilepsy), please tell your doctor as they might interact with your treatment with 

Cystadane. 

  

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. Your doctor will decide if you can use this 

medicine during pregnancy and breast-feeding. 

 

Driving and using machines 
Cystadane has no or negligible influence on the ability to drive and use machines.  

 

 

3. How to take Cystadane 
 

The use of this medicine will be supervised by a doctor experienced in the treatment of patients with 

homocystinuria. 

Always take this medicine exactly as your doctor or pharmacist has told you. Check with your doctor 

or pharmacist if you are not sure.  

 

The recommended dose in children and adults is 100mg/kg/day divided in 2 dosesper day. In some 

patients doses above 200 mg/ kg/day were needed to reach therapeutic goals. Your doctor may adapt 

the dose depending on your laboratory values.  

 

You will therefore need regular blood tests to determine the correct daily dose. 

 

You should take Cystadane orally (by mouth). 

To measure the dose:  

 shake the bottle lightly before opening 

 take the correct measuring spoon:  

 the small green spoon measures  100 mg of betaine anhydrous powder;  

 the middle size blue spoon measures  150 mg of betaine anhydrous powder ; 

 the large pink spoon measures 1 g of betaine anhydrous powder. 

 take a heaped spoonful of powder out of the bottle 

 pass the flat back of a knife over the top of the spoon 

 the powder left in the spoon is one spoonful 

 take the correct number of spoonfuls of powder from the bottle 
 

Mix the measured dose of powder with water, juice, milk, formula or food until completely dissolved 

and ingest immediately after mixing. 

 

If you take more Cystadane than you should 

If you accidentally take too much Cystadane, talk to a doctor or pharmacist immediately. 

 

If you forget to take Cystadane 
Do not take a double dose to make up for forgotten doses. If you forget to take a dose take it as soon as 

you remember it and continue the next dose as planned. 

 

 

If you stop taking Cystadane 



 

19 

Do not stop the treatment without consulting your doctor. Contact your doctor or pharmacist before 

stopping. 

 

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

 

 

4. Possible side effects 
 

Like all medicines, this medicine can cause side effects, although not everybody gets them. 

 

The most commonly side effect when taking Cystadane which may affect more than 1 in 10 people 

(frequency very common) is elevated levels of methionine in the blood.  

Methionine level can be related to swelling in the brain (cerebral swelling), which may affect up to 1 

in 100 people (frequency uncommon). If you experience morning headaches with vomiting and/or 

visual changes, contact immediately your doctor (they could be signs of a swelling in the brain). 

 

Gastrointestinal disorders like diarrhoea, nausea, vomiting, stomach discomfort and inflammation of 

the tongue may occur uncommonly (may affect up to 1 in 100 people).  

Other uncommon side effects (may affect up to 1 in 100 people) may include decreased appetite 

(anorexia), agitation, irritability, hair loss, hives, skin odour abnormal, lack of control over passing 

urine (urinary incontinence). 

 

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 Cystadane 

 

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 bottle label and the carton after 

EXP. The expiry date refers to the last day of that month. 

 

Do not store above 25C. 

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

After the first opening of the bottle, the medicine should be used within 3 months. 
 

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. 

 

 

6. Contents of the pack and other information 

 

What Cystadane contains 

- The active substance is betaine anhydrous. 1 g of oral powder contains 1 g of betaine anhydrous.  
- There is no other ingredient. 
 

What Cystadane looks like and contents of the pack 

Cystadane is a white crystalline free flowing powder. It is presented in bottles with child resistant 

closures. Each bottle contains 180 g of powder. Each carton contains one bottle and three measuring 

spoons. 



 

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Marketing Authorisation Holder 

Recordati Rare Diseases 

Immeuble “Le Wilson” 

70, avenue du General de Gaulle  

F-92 800 Puteaux 

France 

 

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 medicine, please contact the local representative of the Marketing 

Authorisation Holder. 

 

Belgique/België/Belgien 

Recordati  

Tél/Tel: +32 2 46101 36 

Belgique 

Lietuva 

Recordati AB. 

Tel: + 46 8 545 80 230 

Švedija 

 

България 

Recordati Rare Diseases 

Tel: +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  



 

21 

  

Ελλάδα 

Recordati Rare Diseases 

Τηλ: +33 (0)1 47 73 64 58 

Γαλλία 

 

Österreich 

Recordati Rare Diseases Germany GmbH 

Tel: +49 731 140 554 0 

Deutschland 

 

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 

United Kingdom 

Recordati Rare Diseases UK Ltd. 

Tel: +44 (0)1491 414333 

Zviedrija 

 

 

This leaflet was last revised in  
 

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