PBAC's latest decision on Vosoritide: Recommended with restriction (2022). Considered for Treatment of patients with achondroplasia whose epiphyses are not closed.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2022
Submissions
1
2022 → 2022
Eligible population
Patients with achondroplasia whose epiphyses are not closed (age agnostic, but clinical evidence primarily from patients ≥5 years and some data from patients 6 months to <5 years).
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Annual growth velocity
Pivotal trial size
75 patients
Key trials
Study 111-301, Study 111-206
Comparator
Best supportive care (BSC)
Economic model
CUA
ICER note
ICER not stated in the public document. Economic model exists but values not disclosed in PSD.