Initial Treatment
Lumasiran may be considered medically necessary if all of the following conditions are met:
Diagnosis of primary hyperoxaluria type 1 confirmed by identification of biallelic pathogenic variants in alanine:glyoxylate aminotransferase (AGT or AGXT) gene OR liver biopsy demonstrating AGT deficiency.
Presence of 1 of the following clinical signs or symptoms of primary hyperoxaluria type 1:
Elevated urine oxalate excretion (body surface area-normalized daily urine oxalate excretion output ≥0.7 mmol/1.73 m2)
Elevated plasma oxalate concentration >20 μmol/L or >1.76 mg/L
Urine oxalate excretion:creatinine ratio above age-specific upper limit of normal.
Individual has not received a liver transplant.
Prescribed by or in consultation with a nephrologist, urologist, geneticist, or any healthcare provider with expertise in treating primary hyperoxaluria type 1.
Initial authorization period is for 6 months.
Continuation of Treatment
Incremental reauthorization for lumasiran may be considered medically necessary if the following conditions are met:
Individual continues to meet the initial treatment criteria cited above.
Documented evidence to support clinically meaningful response to therapy from pre-treatment baseline (eg, decreased urinary oxalate concentrations, decreased urinary oxalate:creatinine ratio, decreased plasma oxalate concentrations, improvement, stabilization or slowed worsening of nephrocalcinosis, renal stone events, renal impairment or systemic calcinosis).
Does not exceed United States Food and Drug Administration approved maintenance dose.
Reauthorization period is for 12 months.
Lumasiran is considered investigational when the above criteria are not met.
Lumasiran is considered investigational for all other indications.
