Pegcetacoplan (Empaveli)

Key Messages

• Canada’s Drug Agency (CDA-AMC) recommends that Empaveli be reimbursed by public drug plans for patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) to reduce proteinuria if certain conditions are met.
• The Canadian Drug Expert Committee (CDEC) determined that it is uncertain whether Empaveli demonstrates acceptable clinical value versus placebo in patients aged 12 years and older with C3G or primary IC-MPGN to reduce proteinuria. Evidence from a clinical trial showed that 26 weeks of treatment with Empaveli, when added to supportive care, reduced protein in the urine and may help slow worsening kidney function compared with supportive care alone. However, the evidence for Empaveli is uncertain because the study was small and did not show whether the treatment improves important long-term outcomes like kidney failure, heart problems, or survival.
• Empaveli should only be covered for patients aged 12 years and older with a confirmed diagnosis of C3G or primary IC-MPGN based on kidney biopsy, who have an estimated glomerular filtration rate (eGFR) of at least 30 mL/min/1.73 m2, and substantial proteinuria. Empaveli should not be used in patients with transplant rejection, disease caused by another condition, or severe kidney scarring (greater than 50% global glomerulosclerosis or interstitial fibrosis on kidney biopsy).
• Empaveli should only be reimbursed if prescribed by a kidney specialist experienced in managing C3G or primary IC-MPGN and the cost of Empaveli is reduced. Empaveli should be initially covered for 6 months and may be continued if the patient shows a meaningful response (such as reduced protein in the urine with stable kidney function, or clear removal of C3 build-up in the kidneys, as seen on a follow-up kidney biopsy), with reassessment at least every year. Important budget impact considerations must be addressed for health systems to be able to adopt Empaveli.