Velmanase AlfaLAMZEDE
Enzyme replacement therapy for the treatment of non-neurological manifestations in patients with alpha-mannosidosis.
Decisions on record
- Meeting Dec 2025 Recommended Alpha-mannosidosis (AM) no PSD
- Meeting Jul 2025 Deferred Non-neurological manifestations of alpha-mannosidosis
Access path
- TGA registered · LAMZEDE
TGA label narrower than the PBS population
- Dec 2025Recommended · restricted
Comparator changed: best supportive care → best supportive care (BSC)
- Dec 2025Deferred
Comparator changed: best supportive care → best supportive care (BSC)
From the public summary
The PBAC deferred making a recommendation for the listing of velmanase alfa for the treatment of non-neurological manifestations of alpha-mannosidosis (AM), to allow for further discussion to better clarify the benefits of treatment and associated cost-effectiveness, and to further develop the restriction.PSD · Jul 2025
However, the PBAC considered that whilst the clinical data demonstrated that velmanase alfa is biochemically effective for reducing serum oligosaccharides, further information from the Sponsor regarding the clinical and patient-relevant benefits of treatment would be informative. The PBAC considered that the proposed cost of velmanase alfa was very high and uncertain as it was dependent on weight-based dosing and the duration of therapy.PSD · Jul 2025
The submission presented a cost-utility analysis comparing velmanase alfa plus BSC to BSC alone for the treatment of patients with AM. Table 12 presents a summary of the model structure and rationale. 22PSD · Jul 2025
OFFICIAL Public Summary Document– July 2025 PBAC Meeting with Corrigendum and December 2025 Addendum Table 12: Key components of the economic evaluation Component Description Justification/comments Treatment Velmanase alfa and BSC vs BSC - Lifetime (until death) in the base-case in the The time horizon in the base case exceeded the average Time horizon model base case versus 4 years (48 month) in life expectancy for patients with AM of 40 years.PSD · Jul 2025
The submission described velmanase alfa as superior in terms of effectiveness compared with BSC and inferior in terms of safety compared to BSC.PSD · Jul 2025
The Sub-Committees considered that the claim of superior effectiveness to BSC was reasonably supported by the evidence presented in the submission; however, noted that the following issues should be considered:PSD · Jul 2025
The PBAC noted and welcomed the input from individuals (1), and consumer organisation The International Society for Mannosidosis and Related Diseases (ISMRD) via the Consumer Comments facility on the PBS website.PSD · Jul 2025
The PBAC welcomed the input from the ISMRD that highlighted the devastating impact of alpha-mannosidosis, which include intellectual disabilities, skeletal abnormalities, ataxia, muscle weakness, mobility issues, seizures and psychiatric symptoms (amongst others) and highlighted the urgent clinical need for velmanase alfa to be made available to Australian patients.PSD · Jul 2025
Cost-effectiveness
ICER not stated in the public text; economic model reliability was questioned by PBAC due to uncertainty in clinical data
The PBAC considered that velmanase alfa was not cost effective, noting the cost per patient per year was substantially higher than that for previously recommended treatments for ultra-rare diseases funded on the PBS with benefits which are likely similar in terms of clinical impact. PBAC · 2025
Decision context
PopulationPatients with non-neurological manifestations of alpha-mannosidosis, diagnosed by leukocyte-based assay with or without genetic testing.
Risk sharingPBAC considered that a risk sharing arrangement (RSA) would be an appropriate way to manage potential use outside the restrictions and higher than predicted average doses (as dosing is weight based).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Dec 2025 | Recommended · restricted | best supportive care | — | RCT · Surrogate |
| Dec 2025 | Deferred | best supportive care (BSC) | — | RCT |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| rhLAMAN-08 | Ph 2 | 5 | Safety and tolerability of velmanase alfa as per Adverse events | completed |
Consumer voice
A parent of a child with alpha-mannosidosis and the International Society for Mannosidosis and Related Diseases (ISMRD) provided input highlighting the severe impact of the disease on everyday life, the ineffectiveness of current symptomatic treatments, and the urgent clinical need for velmanase alfa. They noted significant positive effects observed in Europe and the United States, with improvemen
the severe impact of disease on everyday life, the mental and physical impacts of the condition, and stated that current symptomatic treatments had not been effective. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to aged 6+ and first-line use; TGA label has no age or line-of-therapy restrictions.