Cipaglucosidase Alfa With Miglustat
Treatment of adults with late-onset Pompe disease (LOPD), a rare autosomal recessive glycogen storage disorder caused by GAA gene mutations leading to progressive metabolic myopathy and respiratory dysfunction.
Decision on record
- Meeting Mar 2025 Not recommended Late onset Pompe disease
From the public summary
7.1 The PBAC did not recommend the requested Section 100 (Highly Specialised Drugs Program) listing of cipaglucosidase alfa and miglustat (CIPAMIG) for the treatment of adults with late-onset Pompe disease (LOPD). The PBAC considered treatment with CIPAMIG for LOPD was likely non-inferior to alglucosidase alfa (ALGLU) and avalglucosidase alfa (AVAL).PSD · Mar 2025
7.2 The PBAC noted that the submission had stated its objective was to seek listing CIPAMIG on the Life Saving Drugs Program (LSDP) alongside the existing enzyme replacement therapies (ERTs) for the treatment of LOPD.PSD · Mar 2025
6.60 The submission presented a cost-minimisation approach (CMA), on the basis that CIPAMIG is non-inferior to ALGLU and AVAL. The submission stated that ‘in November 2021 AVAL was recommended on a cost-minimisation basis vs ALGLU and the resulting AVAL price was a cost-minimised price in adults with LOPD’, based on Table 20 in the November 2021 AVAL PSD.PSD · Mar 2025
Table 20 in the AVAL PSD was the estimated cost per patient per year for AVAL, not a cost-minimisation analysis. The submission presented an analysis of CIPAMIG vs AVAL in adults with the assumption that the cost of ALGLU is the same or higher. The key components of the analysis are shown in Table 10.PSD · Mar 2025
6.55 The clinical claims were that in adults with LOPD, CIPAMIG was "at least non-inferior" in efficacy and non-inferior in safety to ALGLU and AVAL.PSD · Mar 2025
6.56 With regard to efficacy, the evaluation noted this claim was not adequately supported for the comparison with either ALGLU or AVAL. In relation to ALGLU, the observed changes in % predicted FVC and 6MWD in PROPEL were highly variable, with some patients improving significantly and others deteriorating significantly, so that small differences in mean change were difficult to interpret, and, if there is a short-term treatment-switching effect, may not …PSD · Mar 2025
6.2 The PBAC noted and welcomed the input from individuals (16), and organisations (1) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with CIPAMIG including increased quality of life, improvements in respiratory function and muscle strength.PSD · Mar 2025
6.3 The Australian Pompe Association (APA) expressed its support for making CIPAMIG available to patients with Pompe disease. The APA highlighted that CIPAMIG may provide an alternative treatment for patients who have or may experience sensitivity to existing treatments.PSD · Mar 2025
Cost-effectiveness
Cost-minimisation approach; ICER not modelled as submission requested listing on Life Saving Drugs Program (LSDP) rather than PBS.
Decision context
PopulationAdults aged ≥18 years with late-onset Pompe disease who are not on long-term invasive ventilation for respiratory failure, without severe comorbidities, non-smokers, presenting with FVC <80% predicted (supine and erect) or sleep disordered breathing or significant muscular weakness, and not currently on alglucosidase alfa or avalglucosidase alfa.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2025 | Not recommended | alglucosidase alfa (ALGLU) and avalglucosidase alfa (AVAL) | — | RCT · Non-inferiority in efficacy and safety |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| PROPEL | Ph 3 | 125 | Change From Baseline to Week 52 in 6 Minute Walk Distance (6MWD) | completed |
Consumer voice
Consumers and the Australian Pompe Association expressed support for CIPAMIG, highlighting benefits including improved quality of life, respiratory function, and muscle strength, as well as the advantage of home-based infusions to reduce travel burden for patients in remote areas. The APA noted CIPAMIG may provide an alternative for patients with sensitivity to existing treatments.
The comments described a range of benefits of treatment with CIPAMIG including increased quality of life, improvements in respiratory function and muscle strength. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to adults not on prior ERT, with specific FVC/respiratory thresholds, excluding invasive ventilation and severe comorbidities; TGA label unrestricted.