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Sapropterin Dipharma is a brand name for Sapropterin. The medicine, its uses, side effects and dosage are the same regardless of brand.
Used for: Sapropterin Dipharma is indicated for the treatment of hyperphenylalaninaemia (HPA) in adults and paediatric patients of all ages with phenylketonuria (PKU) who have been shown to be responsive to such treatment (see section 4.2). Sapropterin Dipharma is also indicated for the treatment of hyperphenylalaninaemia (HPA)…
Verbatim from this product's EMA label. Tap a section to expand.
Treatment with sapropterin dihydrochloride must be initiated and supervised by a physician experienced in the treatment of PKU and BH4 deficiency. Active management of dietary phenylalanine and overall protein intake while taking this medicinal product is required to ensure adequate control of blood phenylalanine levels and nutritional balance.
1). Posology PKU The starting dose of sapropterin dihydrochloride in adult and paediatric patients with PKU is 10 mg/kg body weight once daily. The dose is adjusted, usually between 5 and 20 mg/kg/day, to achieve and maintain adequate blood phenylalanine levels as defined by the physician.
BH4 deficiency The starting dose of sapropterin dihydrochloride in adult and paediatric patients with BH4 deficiency is 2 to 5 mg/kg body weight total daily dose. Doses may be adjusted up to a total of 20 mg/kg per day. 3 Sapropterin Dipharma is provided as 100 mg tablets.
The calculated daily dose based on body weight should be rounded to the nearest multiple of 100. For instance, a calculated dose of 401 to 450 mg should be rounded down to 400 mg corresponding to 4 tablets. A calculated dose of 451 mg to 499 mg should be rounded up to 500 mg corresponding to 5 tablets.
Dose adjustment Treatment with sapropterin may decrease blood phenylalanine levels below the desired therapeutic level. Adjustment of the sapropterin dihydrochloride dose or modification of dietary phenylalanine intake may be required to achieve and maintain blood phenylalanine levels within the desired therapeutic range.
Blood phenylalanine and tyrosine levels should be tested, particularly in the paediatric population, one to two weeks after each dose adjustment and monitored frequently thereafter, under the direction of the treating physician. If inadequate control of blood phenylalanine levels is observed during treatment with sapropterin dihydrochloride, the patient’s adherence to the prescribed treatment, and diet, should be reviewed before considering an adjustment of the dose of sapropterin.
Discontinuation of treatment should be done only under the supervision of a physician. More frequent monitoring may be required, as blood phenylalanine levels may increase. Dietary modification may be necessary to maintain blood phenylalanine levels within the desired therapeutic range.
Summary of the safety profile Approximately 35% of the 579 patients aged 4 years and over who received treatment with sapropterin dihydrochloride (5 to 20 mg/kg/day) in the clinical trials for sapropterin experienced adverse reactions.
The most commonly reported adverse reactions are headache and rhinorrhoea. In a further clinical trial, approximately 30% of the 27 children aged below 4 years who received treatment with sapropterin dihydrochloride (10 or 20 mg/kg/day) experienced adverse reactions.
The most commonly reported adverse reactions are “amino acid level decreased” (hypophenylalaninaemia), vomiting and rhinitis. Tabulated list of adverse reactions 9 In the pivotal clinical trials and in the post-marketing experience for sapropterin, the following adverse reactions have been identified.
The following definitions apply to the frequency terminology used hereafter: 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 available data).
Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness.
Immune system disorders Not known:
Hypersensitivity reactions (including serious allergic reactions) and rash Metabolism and nutrition disorders Common: Hypophenylalaninaemia Nervous system disorders Very common: Headache Respiratory, thoracic and mediastinal disorders Very common: Rhinorrhoea Common: Pharyngolaryngeal pain, nasal congestion, cough Gastrointestinal disorders Common: Diarrhoea, vomiting, abdominal pain, dyspepsia, nausea Not known: Gastritis, oesophagitis Paediatric population Frequency, type and severity of adverse reactions in children were essentially similar to those in adults.
Dietary intake Patients treated with sapropterin dihydrochloride must continue a restricted phenylalanine diet and undergo regular clinical assessment (such as monitoring of blood phenylalanine and tyrosine levels, nutrient intake, and psycho- motor development).
Low blood phenylalanine and tyrosine levels Sustained or recurrent dysfunction in the phenylalanine-tyrosine-dihydroxy-L-phenylalanine (DOPA) metabolic pathway can result in deficient body protein and neurotransmitter synthesis. Prolonged exposure to low blood phenylalanine and tyrosine levels during infancy has been associated with impaired neurodevelopmental outcome.
Active management of dietary phenylalanine and overall protein intake while taking sapropterin dihydrochloride is required to ensure adequate control of blood phenylalanine and tyrosine levels and nutritional balance. Health disturbances Consultation with a physician is recommended during illness as blood phenylalanine levels may increase.
Convulsions disorders Caution should be exercised when prescribing sapropterin dihydrochloride to patients receiving treatment with levodopa. 5). Discontinuation of treatment Rebound, as defined by an increase in blood phenylalanine levels above pre-treatment levels, may occur upon cessation of treatment.
Sodium content This medicinal product contains less than 1 mmol sodium (23 mg) per tablet, that is to say essentially ‘sodium-free’.
1.
Not medical advice. Always read the patient information leaflet and follow your prescriber or pharmacist.
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Determination of response It is of primary importance to initiate treatment as early as possible to avoid the appearance of non- reversible clinical manifestations of neurological disorders in paediatric patients and cognitive deficits and psychiatric disorders in adults due to sustained elevations of blood phenylalanine.
Response to this medicinal product is determined by a decrease in blood phenylalanine. Blood phenylalanine levels should be checked before administering sapropterin dihydrochloride and after 1 week of use at the recommended starting dose.
If an unsatisfactory reduction in blood phenylalanine levels is observed, then the dose can be increased weekly to a maximum of 20 mg/kg/day, with continued weekly monitoring of blood phenylalanine levels over a one month period. The dietary phenylalanine intake should be maintained at a constant level during this period.
A satisfactory response is defined as a ≥ 30 percent reduction in blood phenylalanine levels or attainment of the therapeutic blood phenylalanine goals defined for an individual patient by the treating physician. Patients who fail to achieve this level of response within the described one month test period should be considered non-responsive, these patients should not be treated with sapropterin dihydrochloride and administration of sapropterin dihydrochloride should be discontinued.
Once responsiveness to the medicinal product has been established, the dose may be adjusted within the range of 5 to 20 mg/kg/day according to response to therapy. It is recommended that blood phenylalanine and tyrosine levels be tested one or two weeks after each dose adjustment and monitored frequently thereafter under the direction of the treating physician.
Patients treated with sapropterin dihydrochloride must continue a restricted phenylalanine diet and undergo regular clinical assessment (such as monitoring of blood phenylalanine and tyrosine levels, nutrient intake, and psycho- motor development).
Special populations Elderly Safety and efficacy of sapropterin dihydrochloride in patients above 65 years of age have not been established. Caution must be exercised when prescribing to elderly patients. Renal or hepatic impairment 4 Safety and efficacy of sapropterin dihydrochloride in patients with renal or hepatic insufficiency have not been established.
Caution must be exercised when prescribing to such patients. Paediatric population The posology is the same in adults, children, and adolescents. Method of administration Oral use. Sapropterin Dipharma tablets should be administered with a meal to increase the absorption.
For patients with PKU, Sapropterin Dipharma should be administered as a single daily dose, and at the same time each day preferably in the morning. For patients with BH4 deficiency, divide the total daily dose into 2 or 3 administrations, distributed over the day.
The prescribed number of tablets should be placed in a glass or cup of water or apple juice and stirred until dissolved. It may take a few minutes for the tablets to dissolve. To make the tablets dissolve faster they can be crushed. Small particles may be visible in the solution and will not affect the effectiveness of the medicinal product.
The solution should be drunk within 15 to 20 minutes. Sapropterin Dipharma soluble tablets may also be crushed and then mixed in a small amount of soft foods such as apple sauce or pudding. The prescribed dose of Sapropterin Dipharma soluble tablets dissolved in water as described […]
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.