PBAC's latest decision on Sapropterin dihydrochloride: Recommended with restriction (2011). Considered for Treatment of hyperphenylalaninaemia (HPA) due to phenylketonuria (PKU) in patients who are sapropterin responsive, and treatment for pregnant women with HPA due to PKU or tetrahydrobiopterin (BH4) deficiency.
PBAC outcome
Recommended with restriction
2011
ICER (AUD/QALY)
$200k/QALY
Range: $105k/QALY–$200k/QALY
Submissions
1
first 2011
Submissions
1
2011 → 2011
Eligible population
patients with hyperphenylalaninaemia (HPA) due to phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency who are sapropterin responsive
Therapy area
Metabolic
Evidence base
RCT, Single-arm, Registry
Primary endpoint
Phe levels reduction, PFS
Key trials
PKU-003, PKU-006 Part 2, PKU-001, PKU-004, PKU-007
Comparator
phenylalanine-restricted diet with phenylalanine-free protein supplements (for PKU); prior treatment with sapropterin (for BH4 deficiency)