Sapropterin DihydrochlorideKUVAN
Recommended Metabolic
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.
1
Submissions
2011–11
On the record
$105k–200k
ICER range
1 sourced ICER · 2011
ICER stated
Cost basis
Decisions on record
- Meeting Jul 2022 Recommended Phenylketonuria, hyperphenylalaninemia in adult patients
- Meeting Dec 2020 Recommended Maternal phenylketonuria (MPKU) no PSD
- Meeting Nov 2020 Deferred Maternal phenylketonuria (MPKU)
- Meeting Jul 2019 Recommended Hyperphenylalaninaemia (HPA)
- Meeting Nov 2018 Recommended Hyperphenylalaninemia (HPA)
- Meeting Mar 2018 Deferred Hyperphenylalaninaemia (HPA)
- Meeting Nov 2012 Recommended Hyperphenylalaninaemia
- Meeting Jul 2012 Not recommended Treatment of hyperphenylalaninaemia (HPA) in sapropterin-responsive adult and paediatric patients with phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency.
1 earlier decision
- Nov 2011 Not recommended Treatment of elevated phenylalanine blood levels (rare metabolic disorder)
Access path
- TGA registered · KUVAN
TGA label narrower than the PBS population
- Nov 2011Recommended · restricted
vs phenylalanine-restricted diet with phenylalanine-free…
- PBS listing
RecommendedDeferredNot recommended
Cost-effectiveness
ICER uncertainSurrogate endpoint
Decision context
Populationpatients with hyperphenylalaninaemia (HPA) due to phenylketonuria (PKU) or tetrahydrobiopterin (BH4) deficiency who are sapropterin responsive
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2011 | Recommended · restricted | phenylalanine-restricted diet with phenylalanine-free protein supplements (for PKU); prior treatment with sapropterin (f | $105k–200k | RCT, Single-arm, Registry · Phe levels reduction, PFS |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| PKU-006 Part 2 | Ph 3 | 111 | Tabulation of the Incidence and Frequency of All AEs and SAEs That Occur Throughout the St… | completed |
Similar precedents
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Regulatory · TGA
Label narrower than PBS population — PBAC restricts to sapropterin-responsive patients and adds specific pregnancy indication; TGA label includes all HPA patients regardless of sapropterin responsiveness.