Sebelipase AlfaKanuma
Infantile onset lysosomal acid lipase deficiency (LAL-D), also known as rapidly progressive LAL-D or Wolman disease.
Decision on record
- Meeting Mar 2022 Not recommended Infantile onset lysosomal acid lipase deficiency
Access path
- TGA registered · Kanuma
TGA label narrower than the PBS population
- Mar 2022Not recommended
vs best supportive care (BSC) alone
From the public summary
7.1 The PBAC did not recommend the Section 100 (Highly Specialised Drugs Program) listing of sebelipase alfa for the treatment of infantile onset lysosomal acid lipase deficiency (LAL-D). The PBAC considered sebelipase alfa was an effective treatment for infantile onset LAL-D; however, the incremental cost effectiveness ratio (ICER) for sebelipase alfa compared to best supportive care was extremely high and uncertain.PSD · Mar 2022
7.2 The PBAC noted the consumer comment received for this item was supportive of making sebelipase alfa available for infants with this very rare condition. The PBAC noted the health care professional that provided a consumer comment had also 28PSD · Mar 2022
6.37 The submission presented a cost utility analysis. Table 10 presents a summary of the model structure and rationale.PSD · Mar 2022
6.38 The economic evaluation base case and all the submission’s sensitivity analyses resulted in incremental cost effectiveness ratios (ICER) that were extremely high (> $1,055,000 per quality adjusted life year (QALY)). No plausible sensitivity analyses or alternative base cases could be specified for a conventionally acceptable ICERPSD · Mar 2022
6.32 The submission described sebelipase alfa as superior in terms of effectiveness compared with BSC and non-inferior in terms of safety compared to BSC.PSD · Mar 2022
6.33 The claim of superior effectiveness was reasonably supported by the evidence presented in the submission, and it was likely that patients with infantile onset LAL-D treated with sebelipase alfa would derive a survival benefit compared to BSC alone.PSD · Mar 2022
6.2 The PBAC noted and welcomed the input from a health care professional via the Consumer Comments facility on the PBS website. The comments described the benefits of treatment with sebelipase alfa observed in a patient with infant onset LAL- D and an adolescent patient with CESD. The health care professional suggested that sebelipase alfa may enable patients to become medically stable and suitable for a bone marrow transplant.PSD · Mar 2022
6.61 The submission did not propose a risk sharing arrangement. For more detail on PBAC’s view, see section 7 PBAC outcomePSD · Mar 2022
Cost-effectiveness
No ICER presented in the submission; cost-effectiveness analysis appears not to have been completed or results not reported in the public summary.
Decision context
PopulationInfants with infantile onset lysosomal acid lipase deficiency (LAL-D) with confirmed diagnosis (LAL activity <1%), documented LAL gene abnormalities, and rapid disease progression demonstrated by manifestations such as severe vomiting, diarrhoea, growth failure, hepatosplenomegaly, coagulation disturbance, severe anaemia, or abnormal liver function tests.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2022 | Not recommended | best supportive care (BSC) alone | — | Single-arm · OS |
Consumer voice
A health care professional provided input describing benefits of sebelipase alfa in two patients with LAL-D/CESD, noting it may enable medical stability and suitability for bone marrow transplant, while highlighting concerns about central venous access requirements and potential ADA development limiting long-term use.
The comments described the benefits of treatment with sebelipase alfa observed in a patient with infant onset LAL-D and an adolescent patient with CESD. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to infantile-onset LAL-D with rapid progression; TGA label covers all ages without disease progression criteria.