Avalglucosidase AlfaNexviazyme
Long-term enzyme replacement therapy for the treatment of patients with Pompe disease (infantile-onset, juvenile-onset, and adult-onset Pompe disease with impaired respiratory function, sleep disordered breathing or significant muscle weakness).
Decision on record
- Meeting Nov 2021 Not recommended Pompe disease
Access path
- TGA registered · Nexviazyme
TGA label narrower than the PBS population
- Nov 2021Not recommended
vs alglucosidase alfa
From the public summary
The PBAC did not recommend the requested Section 100 (Highly Specialised Drugs Program) listing of avalglucosidase alfa (AVAL) for the treatment of infantile-onset Pompe disease (IOPD), juvenile-onset Pompe disease (JOPD) or adult-onset Pompe disease (AOPD). The PBAC considered AVAL treatment for late onset Pompe disease (LOPD), including the AOPD and JOPD populations, was likely non-inferior to alglucosidase alfa (ALGLU).PSD · Nov 2021
The PBAC noted the Life Saving Drugs Program (LSDP) Expert Panel suggested nomenclature and considered it would be appropriate to consolidate IOPD and LOPD.PSD · Nov 2021
The submission presented a cost-minimisation approach (CMA) comparing AVAL with ALGLU and cost-utility analysis (CUA) comparing ALGLU (as a proxy for AVAL) with BSC, for LOPD and IOPD.PSD · Nov 2021
CMA – avalglucosidase alfa (AVAL) vs alglucosidase alfa (ALGLU) The submission presented separate CMAs comparing AVAL with ALGLU for LOPD and IOPD patients based on the therapeutic conclusions of non-inferior efficacy and safety.PSD · Nov 2021
Avalglucosidase alfa (AVAL) vs alglucosidase alfa (ALGLU) For the primary clinical claim, the submission described AVAL as non-inferior in patients with LOPD and IOPD in terms of effectiveness and safety to ALGLU. However, the evaluation considered the following issues need to be considered when examining the claim: OS was not measured in COMET, NEO1/NEO-EXT, nor mini-COMET nor in their respective ETP.PSD · Nov 2021
This is different to the requested PBS listing for IOPD, where patients must be aged ≤ 24 months at the time of initial application, and there are no restrictions specifying prior 24PSD · Nov 2021
The PBAC noted and welcomed the input from individuals (25), health care professionals (HCP, 2) and organisations (2) via the Consumer Comments facility on the PBS website. The HCPs described the impacts of Pompe Disease on patients’ quality of life, particularly with weakness and respiratory issues, and highlighted the improvements for patients on treatment in regaining function and being active members of their community.PSD · Nov 2021
The PBAC noted the advice received from the Australian Pompe Association clarifying the potential benefits of using AVAL in clinical practice, in particular the reduced administrative burden with quicker reconstitution time providing more flexibility with appointment times, and home infusions providing less disruption to patients’ lives.PSD · Nov 2021
Cost-effectiveness
Cost minimisation analysis versus alglucosidase alfa; secondary CEA versus placebo presented.
Decision context
PopulationInfantile-onset Pompe disease (aged ≤24 months), juvenile-onset Pompe disease (aged 24 months to <18 years), and adult-onset Pompe disease (aged ≥18 years) with impaired respiratory function (FVC <80% predicted), sleep disordered breathing, or significant muscle weakness.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2021 | Not recommended | alglucosidase alfa | — | Single-arm · FVC % predicted, 6MWT, MIP, MEP |
Consumer voice
Consumer input from 25 individuals, 2 healthcare professionals, and 2 organisations described the significant quality of life impacts of Pompe Disease and highlighted improvements from treatment, with particular emphasis on the benefits of home infusions in reducing administrative burden and improving access for rural and remote patients.
The HCPs described the impacts of Pompe Disease on patients' quality of life, particularly with weakness and respiratory issues, and highlighted the improvements for patients on treatment in regaining function and being active members of their community. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to Pompe disease patients with specific functional impairments (respiratory, sleep, or muscle weakness); TGA label covers all Pompe disease patients ≥1 year.