Source: European Medicines Agency (EU) Revision Year: 2017 Publisher: Leadiant GmbH, Liebherrstr. 22, 80538 Munich, Germany, Telephone: +49 (0)89 5506675 – 0, Fax: +49 (0) 89 55 066 75 25, e-mail: info@leadiantbiosciences.com
Chenodeoxycholic acid is indicated for the treatment of inborn errors of primary bile acid synthesis due to sterol 27 hydroxylase deficiency (presenting as cerebrotendinous xanthomatosis (CTX)) in infants, children and adolescents aged 1 month to 18 years and adults.
Treatment must be initiated and monitored by physicians experienced in the management of CTX or inborn errors of primary bile acid synthesis.
During the initiation of therapy and dose adjustment, serum cholestanol levels and/or urine bile alcohols should be monitored every 3 months till metabolic control and then annually. The lowest dose of chenodeoxycholic acid that effectively reduces the serum cholestanol and/or urine bile alcohols levels to within the normal range should be chosen. Liver function should also be monitored. Concurrent elevation of liver enzymes above normal levels may indicate overdose. After the initiation period, cholestanol, urine bile alcohols and liver function should be determined annually, at a minimum, and the dose adjusted accordingly. Additional or more frequent investigations may need to be undertaken to monitor therapy during periods of fast growth, concomitant disease and pregnancy (see section 4.6).
In case of persistent lack of therapeutic response to chenodeoxycholic acid monotherapy, other treatment options should be considered.
The starting dosage in adults is 750 mg/day, in three divided doses provided that it is sufficient to normalise serum cholestanol and/or urine bile alcohols. The daily dose can be subsequently increased in 250 mg increments to a maximum of 1000 mg/day if the serum cholestanol and/or urine bile alcohols remain elevated.
The starting dose in children is 5 mg/kg/day in three divided doses. Where the dose calculated is not a multiple of 250 mg, the nearest dose below the maximum of 15 mg/kg/day should be selected, provided that is sufficient to normalise serum cholestanol and/or urine bile alcohols.
The safety and efficacy in neonates less than one month of age has not been established. Limited safety data are available (see section 4.8).
Dose adjustment is not necessary.
No data are available for patients with renal impairment. However, these patients should be carefully monitored and the dose titrated individually.
No data are available for patients with hepatic impairment. However, these patients should be carefully monitored and the dose titrated individually.
Chenodeoxycholic acid capsules can be taken without regards to food. The hard capsules should be taken whole with sufficient water at approximately the same time each day. For infants and children who cannot swallow capsules, the capsules may be carefully opened and the content added to sodium bicarbonate solution 8.4%, see section 6.6.
The potential for harm from overdose is considered extremely low, as accumulation of chenodeoxycholic acid is unlikely due to an efficient endogenous mechanism of elimination and excretion.
3 years.
This medicinal product does not require any special storage conditions.
Capsules are packed in polyvinyl chloride (PVC) blisters sealed with aluminium foil and packed in cardboard boxes.
Packs size: 100 capsules.
For children, adolescents and adults who cannot swallow capsules and/or need to take a dose below 250 mg, the capsule may be opened, the contents of added to 25 mL of sodium bicarbonate solution 8.4% (1 mmol/mL) and mixed to produce a suspension containing chenodeoxycholic acid 10 mg/mL.
For infants the capsules may be opened, the contents added to 50 mL of sodium bicarbonate solution 8.4% (1 mmol/mL) and mixed to produce a suspension containing chenodeoxycholic acid 5 mg/mL.
The active substance itself will be dissolved in the sodium bicarbonate solution and it appears as a suspension because not all components of the capsule contents will be dissolved. The suspension is formed quite easily and is ready when there are no visible lumps or powder left.
It is recommended that this suspension is prepared at the pharmacy and instructions given to the parent on how to administer the suspension.
The suspension should be stored in a glass bottle. Do not refrigerate or freeze. The suspension is stable for up to 7 days.
The pharmacy should provide oral dose syringes of appropriate volume and grading for administering the suspension. The correct volumes should preferably be marked on the oral syringe.
The doctor should provide information on the dose to be received according to the weight of the child. The dose range in children is 5-15 mg/kg per day. Further information is provided in the package leaflet under Information for Healthcare Professionals Only.
Any unused product or waste material should be disposed of in accordance with national requirements.
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