PBAC's latest decision on Burosumab: Recommended with restriction (2022). Considered for Treatment of X-linked hypophosphataemia (XLH) in paediatric and adult patients with confirmed PHEX pathogenic variant or clinical/laboratory criteria including hypophosphataemia, radiographic evidence of rickets, elevated FGF-23 levels, and renal phosphate wasting.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
2
first 2021
Submissions
2
2021 → 2022
Eligible population
Paediatric and adult patients with a documented PHEX pathogenic variant or diagnosis of XLH confirmed by hypophosphataemia, radiographic evidence of rickets, elevated FGF-23 levels, and renal phosphate wasting, treated by endocrinologist or nephrologist specialists.
Therapy area
Endocrinology
Line of therapy
Not applicable
Evidence base
RCT
Primary endpoint
Surrogate
Key trials
KRN23-003, CL303
Comparator
Conventional therapy (oral phosphorus and active vitamin D [calcitriol]) in children; mix of conventional therapy and routine symptomatic management in adults
Economic model
CUA
ICER note
ICER values are not stated in the PSD. The economic evaluation was updated with a price reduction, but specific ICER figures are redacted or not published in this public document.
Risk sharing
Risk-sharing arrangement in place — Risk sharing arrangement (RSA) proposed to reimburse the Australian government a percentage of any expenditure on burosumab over and above annual financial caps, calculated based on the resubmission's base case assumptions of eligibility, uptake, and utilisation.
Submission history
Mar 2021: Recommended with restriction · Authority Required
Mar 2022: Recommended with restriction · Authority Required