Current status: Answered by Jenni Minto on 11 March 2026
To ask the Scottish Government what dosage is specified in the national clinical pathway for responsiveness testing with the drug sapropterin in individuals with phenylketonuria (PKU) in Scotland, and whether that pathway aligns with the recommendations of the British Inherited Metabolic Disease Group (BIMDG).
In Scotland, the national clinical pathway for sapropterin use in individuals with phenylketonuria (PKU) applies the UK best-practice consensus guidelines developed by the British Inherited Metabolic Disease Group (BIMDG). This ensures a consistent approach to dosing and assessment of treatment response across the UK’s metabolic services.
The pathway specifies a starting dose of 10 mg per kilogram of body weight per day for the period of responsiveness testing.
The decision to prescribe a medicine, and which medicine to prescribe, is entirely for the clinician in charge of a person’s care, having taken into account their clinical condition, safety and any relevant clinical guidance.