Drug updated on 4/17/2024
| Dosage Form | Injection (intravenous; 10 mg as a lyophilized powder in a single-dose vial) |
| Drug Class | Recombinant human lysosomal alpha-mannosidases |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients.
Summary
- Velmanase alfa-tycv (Lamzede) is approved for the management of non-central nervous system manifestations of alpha-mannosidosis in both adult and pediatric patients. It has demonstrated a robust safety profile, with treatment-emergent adverse events not leading to permanent cessation of therapy.
- Two randomized controlled studies examined the efficacy and safety of velmanase alfa (Lamzede) in treating alpha-mannosidosis, without comparisons to other drugs.
- These studies documented significant reductions in serum oligosaccharide levels as a primary measure of effectiveness, showing Lamzede's capability in decreasing the accumulation of harmful substances in individuals with alpha-mannosidosis.
- Evaluations utilizing the 3-minute stair climb test revealed significant and sustained improvements, illustrating not only biochemical control but also enhanced physical capabilities in patients receiving Lamzede.
- The findings indicate more notable benefits from velmanase alfa treatment in pediatric patients, suggesting that initiating treatment earlier may lead to improved outcomes, particularly in functional measures.
- Long-term data consolidated in one study provides insights into the durability of effects over time for both adults and pediatric populations; it presents maintenance of improvements up to four years, affirming its long-lasting impact, which is essential for a progressive condition.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Lamzede (velmanase alfa-tycv) Prescribing Information. | 2023 | Chiesi Farmaceutici S.p.A., Parma, Italy |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial. | 25Subjects F: 44% M: 56% | 2018 | Journal of Inherited Metabolic Disease |
| Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis. | 33Subjects F: 39% M: 61% | 2018 | Journal of Inherited Metabolic Disease |
Sex Distribution:
F:44%
M:56%
25Subjects
Year:
2018
Source:Journal of Inherited Metabolic Disease
Sex Distribution:
F:39%
M:61%
33Subjects
Year:
2018
Source:Journal of Inherited Metabolic Disease