Information for health professionals supplying Sapropterin for treatment of tetrahydrobiopterin (BH4) deficiency based on clinical and laboratory grounds.
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 (PB065) for a patient starting initial PBS-subsidised treatment with Sapropterin
For more information contact us on the Complex Drug Enquiry line.