Plozasiran (Redemplo)

Key Messages

• Canada’s Drug Agency (CDA-AMC) recommends that Redemplo be reimbursed by public drug plans for familial chylomicronemia syndrome (FCS) if certain conditions are met.
• The Canadian Drug Expert Committee (CDEC) determined that Redemplo demonstrates acceptable clinical value versus placebo in patients with FCS. This determination was enough for CDEC to recommend that Redemplo be reimbursed. Given that Redemplo is expected to be an additive treatment to low-fat diet, acceptable clinical value refers to added value versus diet alone.
• Evidence from a clinical trial showed that Redemplo given for 12 months improved triglyceride levels and rate of acute pancreatitis in adult patients with FCS. The certainty in the results was moderate due to some imprecision as a result of small sample sizes given the rarity of FCS. Results from the trial about health-related quality of life were inconclusive. Harms data were generally comparable between groups, and few patients discontinued from treatment due to adverse events (AEs).
• Redemplo should only be reimbursed for adult patients with FCS in line with the Health Canada indication.
• Redemplo is expected to address some identified unmet clinical needs compared to current standard of care (i.e., off-label therapies). Tryngolza (olezarsen) recently received a positive recommendation from CDA-AMC, and the safety and efficacy of Redemplo compared to Tryngolza is unknown.
• Redemplo should only be reimbursed if patients are diagnosed with FCS based on clinical or genetic assessments as detailed in Table 1, and if the cost of Redemplo is reduced. Because FCS is a rare disease, initiation and management of therapy with Redemplo should be managed by a specialist with expertise in treating FCS. The recommended period for initial reimbursement is 6 months, followed by annual renewals thereafter. Renewal is recommended to be based on an observation of clinical benefit, as demonstrated with a reduction in fasting triglyceride levels, and no signs of significant disease worsening that would indicate poor or nonresponse to treatment (i.e., increased rate of acute pancreatitis as judged by the treating clinician).