PBAC's latest decision on Sapropterin: Noted (2022). Considered for Extension of current PBS listing for hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) to allow adults aged ≥18 years to be eligible for sapropterin responsiveness testing and continuing treatment for those who are sapropterin responsive.
PBAC outcome
Noted
Restricted
ICER (AUD/QALY)
—
ICER not stated
Submissions
6
first 2012
Submissions
6
2012 → 2022
Eligible population
Adults aged ≥18 years with hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) who are sapropterin responsive, with baseline blood phenylalanine level >600 micromole per L.
Therapy area
Metabolic
Line of therapy
Any
Evidence base
RCT
Primary endpoint
Reduction in blood phenylalanine levels by >30% from sapropterin-naïve baseline
Key trials
PKU-001
Comparator
phenylalanine (Phe)-restricted diet
Economic model
CUA
ICER note
ICER previously estimated at >$200,000/QALY in March 2018 submission for patients aged ≥18 years, considered unacceptably high. Current submission's economic model outcome not explicitly stated in the PSD text provided.
ICER (historical)
$45k/QALY–$75k/QALY in an earlier submission (2012) — the latest submission carried no numeric base case. No single PSD states this combined range; see source PSDs.
Risk sharing
Risk-sharing arrangement in place — Special pricing arrangements apply to sapropterin listing as noted in the requested listing table.
Submission history
Jul 2012: Not recommended — ICER $75k/QALY
Mar 2018: Deferred · Authority Required
Nov 2018: Recommended with restriction · Authority Required
Jul 2019: Recommended with restriction · Authority Required
Nov 2020: Recommended with restriction · Authority Required