BurosumabCRYSVITA
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.
Decisions on record
- Meeting Mar 2026 Recommended Tumour induced osteomalacia (TIO) with hypophosphatemia no PSD
- Meeting May 2022 Recommended X-linked hypophosphataemia (XLH) no PSD
- Meeting Mar 2022 Not recommended X-linked hypophosphataemia
- Meeting Mar 2021 Not recommended X-linked hypophosphataemia in paediatric patients
Access path
- TGA registered · CRYSVITA
TGA label narrower than the PBS population
- Mar 2021Not recommended
vs conventional therapy (oral phosphorus and calcitriol)
- Mar 2022Not recommended
high clinical uncertainty regarding long-term outcomes, limited clinical trial data for adolescents aged 13-17 years…
- ↻ resubmittedMay 2022Recommended · restricted
Comparator changed: conventional therapy (oral phosphorus and active vitamin D…
- PBS listing · Authority Required
From the public summary
The PBAC did not recommend burosumab for the treatment of paediatric and adult patients with X-linked hypophosphataemia (XLH). The PBAC noted the high clinical need and strong consumer support for treatments for this condition. However, the PBAC considered that the incremental cost-effectiveness ratio (ICER) was unacceptably high at the proposed price.PSD · Mar 2022
The PBAC recognised the high clinical need for effective XLH treatments and noted the strong consumer support for burosumab describing a range of benefits including the ease of administration compared to currently available therapies and the effectiveness, tolerability and improvements to quality of life associated with burosumab.PSD · Mar 2022
The resubmission presented two separate cost utility analyses comparing burosumab and conventional therapy (oral phosphorus and calcitriol) for the treatment of XLH in children and adults. The paediatric model considered treatment of paediatric patients 30PSD · Mar 2022
Paediatrics The resubmission presented an updated modelled economic evaluation comparing burosumab and conventional therapy for the treatment of XLH in children. The structure of the model was as for the March 2021 submission which was a Markov state transition model with four health states defined by RSS: Mild (RSS of 0.5 or 1.0), Moderate (RSS of 1.5 or 2.0), Severe (RSS of 2.5 or more), Healed (RSS of 0) and Death (to capture background mortality).PSD · Mar 2022
Paediatrics In paediatric patients with XLH, the resubmission described burosumab as superior in terms of effectiveness and superior (and different) in terms of safety compared to conventional therapy comprising oral phosphate and active vitamin D.PSD · Mar 2022
Overall, the PBAC again considered that the clinical claim for effectiveness of burosumab in children was supported by the clinical evidence, but noted the following issues:PSD · Mar 2022
The PBAC noted and welcomed the input from individuals (175), health care professionals (12) and organisations (2) via the Consumer Comments facility on the PBS website. Of the comments from individuals, the PBAC noted several were from individuals living with XLH, or supporting someone with XLH, who had direct experience of using burosumab.PSD · Mar 2022
The PBAC noted the advice received from the Australian and New Zealand Bone and Mineral Society (ANZBMS) and the Australasian Paediatric Endocrine Group (APEG) strongly supporting the use of burosumab in clinical practice. The PBAC specifically noted the advice that the use of burosumab may reverse the serious disabilities associated with XLH and significantly improve quality of life.PSD · Mar 2022
Cost-effectiveness
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.
The PBAC noted the high clinical uncertainty in the model, particularly regarding the reliability of the transition probabilities, the assumptions regarding dose and compliance, and the financial estimates in paediatrics and adults. PBAC · 2022
Decision context
PopulationPaediatric 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.
Risk sharingRisk 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
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| May 2022 | Recommended · restricted | Conventional therapy (oral phosphorus and active vitamin D [calcitriol]) in children; mix of conventional therapy and ro | $355k–455k | RCT · Surrogate |
| Mar 2022 | Not recommended | conventional therapy (oral phosphorus and active vitamin D [calcitriol]) in children; mixture of conventional therapy an | $355k–455k | RCT |
| Mar 2021 | Not recommended | conventional therapy (oral phosphorus and calcitriol) | $655k–755k | RCT · Other |
Codependent tests
| Service | Biomarker | MSAC outcome | Year |
|---|---|---|---|
| FGF-23 testing for X-linked hypophosphatemia (XLH) | FGF-23 (fibroblast growth factor 23) levels | supported | 2024 |
Consumer voice
Consumer input from 175 individuals (many with direct XLH experience or supporting someone with XLH), 12 health care professionals, and 2 organisations described the disabling nature of XLH and highlighted benefits of burosumab including ease of administration, effectiveness, tolerability, and improved quality of life. Professional organisations strongly supported burosumab use, noting it may reve
the disabling nature of XLH, the range of benefits of treatment with burosumab for both paediatric and adult patients including the ease of administration compared to currently available therapies, the effectiveness of the treatment, the tolerability of the treatment and the improved quality of life associated with the treatment Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients with confirmed PHEX variant or specific diagnostic criteria (hypophosphataemia, rickets, elevated FGF23, renal wasting) and specialist oversight; TGA label has no such restrictions.
Listed in law
PBS listings commencing 1 Nov 2022 were made by these 3 determinations under the National Health Act 1953. A determination covers every listing that commenced that day, so this is the legal instrument in force for the date — not a document naming this medicine. Open it and check.
| Determination | Made | Status | |
|---|---|---|---|
| PB 101 of 2022 | 28 Oct 2022 | Repealed | PDF ↗ |
| PB 101 of 2022 | 28 Oct 2022 | Repealed | PDF ↗ |
| PB 99 of 2022 | 28 Oct 2022 | Repealed | PDF ↗ |