Oxlumo

Lumasiran

• Brand(s): Oxlumo
• Dosage Form/Strength: 94.5 mg/0.5 mL Injection
• Effective date: July 29, 2024

Initiation Criteria:

For the treatment of primary hyperoxaluria type 1 (PH1) to reduce the production of endogenous oxalate production in patients who meet all the following criteria:

1. Patients must have a genetically confirmed diagnosis of PH1 as determined by a mutation in the alanine-glyoxylate aminotransferase (AGXT) gene;
   AND
2. Patient has been compliant with Standard of Care (SOC) therapy for PH1 (Note 1) for a duration of 3 to 6 months and meets at least one of the following requirements;
   a) Patient with preserved kidney function where urinary oxalate can be measured but patient has demonstrated that they are unable to normalize 24-hour urine oxalate excretion (Note 2).
   b) Pediatric patient aged 6 years and younger who is noncontinent and who has a high spot oxalate to creatinine (oxalate:creatinine) ratio compared with normal age-based reference ranges
   c) the Patient has end-stage kidney disease and either:
   i) has an estimated glomerular filtration rate (eGFR) less than 30 mL/min/1.73m² and has an increased plasma oxalate level compared with normal laboratory reference ranges, which must be submitted with the request; OR
   ii) is on dialysis and has an increased pre-dialysis plasma oxalate level compared with normal laboratory reference ranges, which must be submitted with the request;
   All requests must include a recent eGFR that is measured at the time of the measurement of urine oxalate levels and/or plasma oxalate levels to ensure an accurate evaluation of the laboratory findings.
3. Initial requests must be provided by a nephrologist or metabolic diseases specialist with experience in the diagnosis and management of PH1.

Include the following clinical details with your request:

• Kidney function and most recent eGFR
• Confirm if patient is on dialysis
• pre-treatment kidney stone history and/or frequency per year. A nil result can be provided.
• Description of systemic oxalosis in organs and tissues, including nephrocalcinosis or confirmation of no systemic deposition.

Discontinuation Criteria

• Treatment with lumasiran should be discontinued if there is evidence of no response or loss of response.
  Response is defined as the below:
  a) For patients in whom urinary oxalate can be measured: Lowering of 24-hour urine oxalate to less than 1.5 times the Upper Limit of Normal (ULN) (Note 2, 3 and 4)
  b) For patients 6 years of age or younger who are noncontinent: Demonstrating a 30% reduction in spot oxalate:creatinine ratio (Note 5 and 6)
  c) For patients with end-stage kidney disease or who are on dialysis: Demonstrating a 15% reduction in plasma oxalate level. (Note 3)
• Treatment with lumasiran should be discontinued if the patient has received a liver transplant with or without a kidney transplant.

Notes:

1. Standard of Care therapy means therapy that includes vitamin B6 (pyridoxine), oral hyperhydration, and crystallization inhibitors, such as citrate supplementation or bicarbonate salts that lower urine acidity, including potassium or sodium citrate and potassium or sodium bicarbonate, and an oxalate-controlled diet used at adequate doses.
2. 24 hour urine oxalate results should be corrected for creatinine or body surface area. Additionally, due to the possibility of individual day-to-day variations in the results, repeated measurement may be provided to ensure appropriate evaluation of baseline values compared with the required response to therapy parameters.
3. Patients with end-stage kidney disease (ESKD) or those who are on dialysis should have pre-dialysis plasma oxalate levels measured instead of urine oxalate. Please submit the plasma oxalate.
4. Evaluate if patient is adherent to standard of care treatment, oxalate controlled dietary restrictions, and determine if eGFR has declined.
5. In patients who are less than 18 years of age, oxalate excretion results should be corrected to a mg per body surface area (BSA) of 1.73 m² to be properly interpreted.
6. Measurement of oxalate:creatinine ratio should be taken at least twice a year to monitor for response to treatment.

Exclusion Criteria for both initial and renewal criteria:

• Patients with Primary hyperoxaluria type 2 (PH2) are not funded.
• Patients with Primary hyperoxaluria type 3 (PH3) are not funded.
• Patients who have received a liver transplant for PH1 are not funded.

Renewal Criteria

Renewals will be considered in patients who do not meet the discontinuation criteria and who continue to demonstrate a response to treatment.

At the time of renewal please submit a recent eGFR along with the biochemical results. Please include clinical details of improvements to kidney function, kidney stone frequency in the past year, and symptoms of systemic oxalosis, as applicable, to demonstrate a response to treatment. You may include consult notes.

Subsequent renewals following the initial prescription can be requested by a pediatrician instead of a nephrologist or metabolic diseases physician.

Approval duration: 12 months

Approved doses:
Loading dose for less than 20 kg patients – up to 6 mg/kg once monthly for 3 doses

Maintenance doses
- Patients less than 10 kg – 3 mg/kg once a month
- Patients 10 kg to less than 20 kg – up to 6 mg/kg once every 3 months.
- Loading dose for patient greater than 20 kg – 3 mg/kg once monthly for 3 doses followed by maintenance doses of up to 3 mg/kg once every 3 months.

EAP Drug Request Form:

Standard Form for EAP Drug Requests