The PBS subsidises sapropterin for patients with tetrahydrobiopterin (BH4) deficiency.
The Schedule of Pharmaceutical Benefits on the PBS website outlines restrictions for prescribing BH4 deficiency.
All patients must have:
- hyperphenylalaninaemia (HPA) due to tetrahydrobiopterin (BH4) deficiency, and
- documented BH4 deficiency using tests for BH4 loading and/or urine pterin metabolites, blood spot dihydropteridine (DHPR) and have cerebrospinal fluid neurotransmitter metabolites measured
Schedule item details
Dose - Sapropterin is available as 100 mg soluble tablet. The starting dose is 2 to 5 mg/kg body weight once daily.
Patients must meet the relevant criteria as indicated in the restrictions and be eligible for the Pharmaceutical Benefits Scheme.
A copy of the relevant pathology reports confirming the presence of hyperphenylalaninaemia (HPA) due to tetrahydrobiopterin (BH4 deficiency) documented BH4 deficiency using tests for:
- BH4 loading and/or urine pterin metabolites
- blood spot dihydropteridine reductase (DHPR), and
- cerebrospinal fluid neurotransmitter metabolites
Lodging an application
Send all written applications to Complex drugs address on the contact us page.
- lodge the Sapropterin for BH4 deficiency Initial and continuing PBS authority application form for a patient starting initial PBS-subsidised treatment with Sapropterin
For more information contact us on the Complex Drug Enquiry line.
Page last updated: 2 May 2019