Coverage Policy Manual
Policy #: 2021032
Category: Pharmacy
Initiated: September 2021
Last Review: June 2022
  Lumasiran (e.g., Oxlumo)

Description:
Primary hyperoxalurias are a group of rare genetic diseases. There are 3 subtypes each resulting in the overproduction of oxalate by the liver. Type 1 is the most common type, which accounts for approximately 80% of cases and occurs as a result of a genetic defect in the alanine:glyoxylate aminotransferase (AGXT) gene that encodes the enzyme alanine glyoxylate aminotransferase. Defect in the enzyme results in overproduction of oxalate, which leads to deposition of calcium oxalate crystals in the kidneys and urinary tract. The result is the formation of painful and recurrent nephrolithiasis (renal stones), nephrocalcinosis, and renal failure. Compromised renal function exacerbates the disease as the excess oxalate can no longer be effectively excreted, resulting in subsequent accumulation and crystallization in bones, eyes, skin, and heart, leading to severe illness and death. Lumasiran is a subcutaneously administered RNAi therapeutic that silences the HAO1 gene, which encodes for a glycolate oxidase enzyme. By silencing the HAO1 gene, levels of glycolate oxidase are depleted decreasing production of oxalate; the metabolite that directly contributes to the pathophysiology of primary hyperoxaluria type 1.
 
Regulatory Status
 
On November 23, 2020, lumasiran (e.g., Oxlumo) was approved by the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients.
 
Coding
C9074 Injection, lumasiran, 0.5 mg
J0224 Ijection, lumasiran, 0.5 mg; effective 7/1/2021

Policy/
Coverage:
Effective September 15, 2021, Prior Approval is required for Lumasiran.
 
The initial use of this drug requires documentation of direct physician involvement and signature in the ordering and evaluation as documented in the medical records submitted for prior approval. Concurrent review will require continued evidence of appropriate physician involvement.  
 
The Step Therapy Medication Act is applicable to fully-insured (Arkansas Blue Cross, Health Advantage, and Exchange) and specified governmental (ASE/PSE and ASP) health plans. The law is not applicable to FEP or self-insured ERISA groups (including but not limited to Walmart, Tyson or other Blue Advantage groups). Initial approval for exigent request is 28 days. Otherwise, initial approval for standard review is up to 1 year.
 
Meets Primary Coverage Criteria Or Is Covered For Contracts Without Primary Coverage Criteria
The use of Lumasiran meets member benefit certificate primary coverage criteria that there be scientific evidence of effectiveness for the treatment of primary hyperoxaluria type 1 when ALL of the following are met:
 
INITIAL APPROVAL STANDARD REVIEW for up to 12 months:
    1. 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
    2. Presence of 1 of the following clinical signs or symptom of primary hyperoxaluria type 1 (Cochat 2012; Milliner 2005):
a.  Elevated urine oxalate excretion (body surface area-normalized daily urine oxalate excretion output 0.7 mmol/1.73 m2)
b.  Elevated plasma oxalate concentration > 20 μmol/L or > 1.76 mg/L
c.  Urine oxalate excretion: creatinine ratio above age-specific upper limit of normal
3.  Individual has not received a liver or kidney transplant (Garrelfs, 2021)
4.  The estimated glomerular filtration rate is > 30 mL/min/1.73m2 (Cochat 2012; Milliner 2005)
5.  Prescribed by or in consultation with a nephrologist, urologist, geneticist, or any healthcare provider with expertise in treating primary hyperoxaluria type 1.
6.  Individual is using in combination with high fluid intake (at least 3 liters/1.73 m2 per day) (Cochat 2012; Hoppe 2009)  AND
7.  Individual is using in combination with pyridoxine OR individual has had a trial and inadequate response to pyridoxine, (Cochat 2012; Hoppe 2009)  AND
8.  Initial approval is for 6 months, limited to the United States Food and Drug Administration approved dosing.
 
Continuation of Treatment (Incremental reauthorization for lumasiran for 1 year)
    1. Individual was previously approved for lumasiran based on criteria cited above
    2. Documented evidence to support clinically meaningful response to therapy from pre-treatment baseline (e.g., decreased urinary oxalate concentrations, decreased urinary oxalate:creatinine ratio, decreased plasma oxalate concentrations)
    3. Individual has not received a liver or kidney transplant
    4. Prescribed by or in consultation with a nephrologist, urologist, geneticist, or any healthcare provider with expertise in treating primary hyperoxaluria type 1.
    5. Does not exceed United States Food and Drug Administration approved maintenance dose.
 
Dosage and Administration
The recommended dose of lumasiran is weight-based and given as a subcutaneous injection. All maintenance doses begin 1 month after the last loading dose.
    • For individuals weighing less than 10 kg: Loading dose is 6 mg/kg once monthly for 3 doses followed by a maintenance dose of 3 mg/kg once monthly.
    • For individuals weighing 10 kg to less than 20 kg: Loading dose is 6 mg/kg once monthly for 3 doses followed by a maintenance dose of 6 mg/kg once every 3 months (quarterly)
    • For individuals weighing 20 kg and above: Loading dose is 3 mg/kg once monthly for 3 doses followed by a maintenance dose of 3 mg/kg once every 3 months (quarterly)
 
Please refer to a separate policy on Site of Care or Site of Service Review (policy #2018030) for pharmacologic/biologic medications.
 
Does Not Meet Primary Coverage Criteria Or Is Investigational For Contracts Without Primary Coverage Criteria
 
The use of Lumasiran does not meet member benefit certificate primary coverage criteria that there be scientific evidence of effectiveness for the treatment of any indication or any circumstance other than those outlined above.
 
For members with contracts without primary coverage criteria, the use of Lumasiran is considered investigational.
 
Investigational services are specific contract exclusions in most member benefit certificates of coverage.

Rationale:
This evidence review was created in April 2021 with searches of the PubMed database. The most recent literature update was performed through April 8, 2021.
 
Evidence reviews assess the clinical evidence to determine whether the use of a technology improves the net health outcome. Broadly defined, health outcomes are length of life, quality of life, and ability to function, including benefits and harms. Every clinical condition has specific outcomes that are important to patients and to managing the course of that condition. Validated outcome measures are necessary to ascertain whether a condition improves or worsens; and whether the magnitude of that change is clinically significant. The net health outcome is a balance of benefits and harms.
 
To assess whether the evidence is sufficient to draw conclusions about the net health outcome of a technology, 2 domains are examined: the relevance and the quality and credibility. To be relevant, studies must represent 1 or more intended clinical use of the technology in the intended population and compare an effective and appropriate alternative at a comparable intensity. For some conditions, the alternative will be supportive care or surveillance. The quality and credibility of the evidence depend on study design and conduct, minimizing bias and confounding that can generate incorrect findings. The randomized controlled trial (RCT) is preferred to assess efficacy; however, in some circumstances, nonrandomized studies may be adequate. Randomized controlled trials are rarely large enough or long enough to capture less common adverse events and long-term effects. Other types of studies can be used for these purposes and to assess generalizability to broader clinical populations and settings of clinical practice.
 
Primary Hyperoxaluria Type 1
 
Clinical Context and Therapy Purpose
 
The purpose of lumasiran in individuals who have primary hyperoxaluria type 1 is to provide a treatment option that is an improvement on existing therapies. Potential benefits of this therapy may include the following:
    • A novel mechanism of action or approach that provides an additional treatment option for many individuals who have failed or have yielded a sub-optimal response to existing treatments.
The question addressed in this evidence review is: Does treatment with lumasiran improve the net health outcome in individuals with primary hyperoxaluria type 1 with preserved renal function?
 
The following PICO was used to select literature to inform this review.
 
Populations
 
The relevant population of interest is individuals with primary hyperoxaluria type 1 with preserved renal function.
 
Interventions
 
The therapy being considered is lumasiran, a RNA interference (RNAi) therapy. It silences the HAO1 gene that encodes for the enzyme glycolate oxidase. Decreased production of glycolate oxidase reduces hepatic oxalate production. Treatment is administered in an outpatient setting. The recommended dose of lumasiran is weight-based and given as a subcutaneous injection. All maintenance doses begin 1 month after the last loading dose.
    • For individuals weighing less than 10 kg: Loading dose is 6 mg/kg once monthly for 3 doses followed by a maintenance dose of 3 mg/kg once monthly.
    • For individuals weighing 10 kg to less than 20 kg: Loading dose is 6 mg/kg once monthly for 3 doses followed by a maintenance dose of 6 mg/kg once every 3 months (quarterly)
    • For individuals weighing 20 kg and above: Loading dose is 3 mg/kg once monthly for 3 doses followed by a maintenance dose of 3 mg/kg once every 3 months (quarterly)
 
Comparators
 
The following therapies are currently being used to manage individuals with primary hyperoxaluria type 1.
 
The initial medical management approaches, which are aimed to delay progressive renal decline, include use of pyridoxine, calcium oxalate crystallization inhibitors, hyperhydration, and dietary restrictions. As the disease progresses, individuals may require interventions for renal stone removal, dialysis, and renal/liver transplant. Despite standard maintenance dialysis therapy, plasma oxalate typically exceeds the supersaturation threshold of 30 micromol/L during a substantial amount of time between dialysis treatments, thereby increasing the risk and progression of systemic oxalosis. Liver transplantation is the only curative intervention as it corrects the underlying enzymatic defect due to mutations of the AGXT gene. In patients with significant chronic renal disease, renal transplant may also be required. Currently, multiple transplantation strategies are in use and include combined liver-renal transplantation, sequential liver and renal transplantation, isolated liver transplantation, and isolated renal transplantation. However, the optimal transplantation type or sequence remains uncertain. Complications include those due to immunosuppressive therapy (eg, infections or adverse drug effects), secondary malignancy, and failure of allograft.
 
Study Selection Criteria
 
Methodologically credible studies were selected using the following principles:
    • To assess efficacy outcomes, comparative controlled prospective trials were sought, with a preference for RCTs;
    • In the absence of such trials, comparative observational studies were sought, with a preference for prospective studies.
    • To assess long-term outcomes and adverse events, single-arm studies that capture longer periods of follow-up and/or larger populations were sought.
    • Studies with duplicative or overlapping populations were excluded.
 
Review of Evidence
 
The clinical development program for lumasiran includes 3 prospective trials. These trials enrolled patients with primary hyperoxaluria type 1 with varying levels of renal function, with age groups ranging from infant to adult. ILLUMINATE-A (NCT03681184) enrolled patients aged 6 years and older with an estimated glomerular filtration rate [eGFR] 30 mL/min/1.73 m2, ILLUMINATE-B (NCT03905694) enrolled patients aged less than 6 years old with an eGFR >45 mL/min/1.73 m2 (if 12 months old) and ILLUMINATE-C (NCT04152200) enrolled patients with advanced primary hyperoxaluria type 1 irrespective of age with an eGFR 30 mL/min/1.73 m2. ILLUMINATE-B has not been published and information was obtained from the prescribing label and FDA review documents.
 
Randomized Controlled Trials
 
ILLUMINATE-A is the pivotal phase 3 randomized, double-blind, placebo-controlled trial. The study consists of 2 parts: an initial 6-month, double-blind treatment period followed by a 54-month extension period in which placebo patients had an option to switch to lumasiran. At 6 months, the percent change in 24-hour urinary oxalate excretion from baseline to month 6 in the lumasiran group was -65% compared to -12% in the placebo group, resulting in a between-group least square (LS) mean difference of 53% (95% confidence interval [CI]: 45 to 62; p<0.0001). The proportion of patients who achieved a 24-hour urinary oxalate level at or below the upper limit of normal (ULN) at month 6 was 52% in the lumasiran group versus 0% in the placebo group (p=0.001). The absolute change in 24-hour urinary oxalate levels in the lumasiran group was -1.24 (95% CI, -1.37 to -1.12) compared to -0.27 (95% CI, -0.44 to -0.10) in the placebo group with a difference of -0.98 (95% CI, -1.18 to -0.78) mmol/24 hr/1.73 m2. Urinary oxalate excretion is a surrogate health outcome and, while it is directly related to the pathophysiology of the disease, ILLUMINATE-A was not powered to assess hard endpoints associated with hyperoxaluria such as renal stones, nephrocalcinosis, and renal failure. Renal stone events was a composite outcome and included at least 1 of the following: visit to healthcare provider because of a renal stone, medication for renal colic, stone passage, or macroscopic hematuria due to a renal stone. Overall, 8 patients (31%) in the lumasiran group and 3 (23%) in the placebo group experienced stone events during the 6-month, placebo-controlled period (13 vs 4 stone events, respectively). Evidence of a treatment effect on kidney stone events was not expected given that calcium oxalate stones are slow to form and pass. The proportion of patients with self-reported stone events at baseline was greater in the lumasiran group versus placebo (89% vs 77%).
 
Single Arm Prospective Trials
 
ILLUMINATE-B is the pivotal single-arm trial in infants and children younger than 6 years. The study consists of 2 parts: (a 6 month primary analysis period followed by a 54 month extension period). At 6 months, the % change in spot urinary oxalate:creatinine ratio from baseline to month 6 was -72% in lumasiran-treated patients. When stratified by weight, the percent reduction was 84%, 67%, and 71% among patients <10 kg (n=3), 10 to <20 kg (n=11), and 20 kg (n=2), respectively. While ILLUMINATE-B lacked a concurrent control, the magnitude of reduction in urinary oxalate and time course were generally consistent with the findings in ILLUMINATE-A.
 
Safety
 
A safety analysis included pooled data from 77 patients (including 56 pediatric patients) from placebo-controlled and open-label clinical studies. Patients ranged in age from 4 months to 61 years at first dose. The median duration of exposure was 9.1 months (range, 1.9 to 21.7 months). Overall, 58 patients were treated for at least 6 months, and 18 patients for at least 12 months. In ILLUMINATE-A, the most common (20%) adverse reaction reported was injection site reaction. Injection site reactions occurred throughout the study period and included erythema, pain, pruritus, and swelling. These symptoms were generally mild and resolved within 1 day of the injection and did not lead to discontinuation of treatment. The safety profile in ILLUMINATE-B was similar to that seen in ILLUMINATE-A. As with all oligonucleotides, there is a concern for development of anti-drug antibodies that may neutralize the therapeutic effect of a drug. As per FDA data analysis from across all clinical studies in the lumasiran development program, including patients with primary hyperoxaluria type 1 and healthy volunteers dosed with lumasiran, 6 of 100 (6%) lumasiran-treated individuals with a mean follow-up duration of 8.9 months tested positive for anti-drug antibodies. No clinically significant differences in the safety, pharmacokinetic, or pharmacodynamic profiles of lumasiran were observed in patients who tested positive for an anti-lumasiran antibody.
 
The purpose of limitations tables is to display notable limitations identified in each study. This information is synthesized as a summary of the body of evidence following each table and provides the conclusions on the sufficiency of the evidence supporting the position statement. The major limitation is the lack of data on clinical outcomes, such as nephrolithiasis and related complications and loss of kidney function, and patient reported outcomes such as quality of life. Given the rarity of the disease and its slow progression, it would be challenging to detect treatment effects on clinical events in a clinical trial. Use of urinary oxalate as a surrogate for clinical outcomes in the pivotal trials can be justified based on the knowledge of the pathophysiology of the disease and the causal role of urinary oxalate in kidney stone formation, nephrocalcinosis, and loss of kidney function. Epidemiologic data demonstrates an association between urinary oxalate and loss of kidney function, particularly in patients with high levels of urinary oxalate. Further observational data from patients treated with pyridoxine or a liver transplant show associations between reductions in urinary oxalate and preservation of kidney function. Further, the consistency and size of treatment effect (more than half of patients receiving lumasiran achieved normal urinary oxalate levels at 6 months of treatment in ILLUMINATE-A) in clinical trials are indicative of the potential for a clinical benefit over the long term. Lastly, while lumasiran was generally well-tolerated in ILLUMINATE-A and -B, the safety database was small and limited in duration.
 
Section Summary: Primary Hyperoxaluria Type 1
 
The evidence for lumasiran for individuals with primary hyperoxaluria with preserved renal function consists of 1 phase 3 RCT (ILLUMINATE-A) in patients 6 years and older and 1 single arm prospective study (ILLUMINATE-B) in patients 6 years and younger. In both studies, patients with preserved renal function were enrolled (eGFR >30 mL/min/1.73 m2). In ILLUMINATE-A, 39 patients were randomized 2:1 to lumasiran or placebo for 6 months. The primary endpoint was the percent change in 24-hour urinary oxalate excretion from baseline to month 6. The percent reduction in 24-hour urinary oxalate from baseline to month 6 was -65% and -12% in the lumasiran and placebo group, respectively, with a between-group mean difference of 53% (95% CI: 45 to 62%; p<0.0001). A similar effect was seen in patients with high baseline urinary oxalate values, and approximately half of patients receiving lumasiran achieved normal urinary oxalate values by month 6. In ILLUMINATE-B, 18 patients were treated with lumasiran. The primary endpoint was the percent change in spot urinary oxalate-to-creatinine ratio from baseline to month 6. Lumasiran demonstrated a percent reduction in spot urinary oxalate-to creatinine ratio from baseline of -71% (95% CI -77 to -65). The magnitude of the reduction and the time course were consistent with findings in ILLUMINATE-A. The major limitation is the lack of data on clinical outcomes such as renal stones, nephrocalcinosis, and renal failure as both trials were not powered to assess these clinical endpoints. However, use of urinary oxalate as a surrogate for clinical outcomes in the pivotal trials may be justified based on the knowledge of the pathophysiology of the disease and the causal role of urinary oxalate in kidney stone formation, nephrocalcinosis, and loss of kidney function.
Further, the consistency and size of treatment effect (more than half of patients receiving lumasiran achieved normal urinary oxalate levels at 6 months of treatment in ILLUMINATE-A) in clinical trials are indicative of the potential for a clinical benefit over the long term. Lastly, while lumasiran was generally well-tolerated in ILLUMINATE-A and -B, the safety database was small and limited in duration. The most common treatment-related adverse events were injection site reactions, which were mild and transient and included erythema, pain, pruritus, or swelling at the injection site.
 
Summary of Evidence
 
For individuals with primary hyperoxaluria type 1 with preserved kidney function, the evidence includes 1 phase 3 RCT (ILLUMINATE-A) in patients 6 years and older and 1 single arm prospective study (ILLUMINATE-B) in patients 6 years and younger. Relevant outcomes are symptoms, quality of life, disease-specific survival, change in disease status, treatment-related morbidity, and treatment-related mortality. In both studies, patients with preserved renal function were enrolled (eGFR >30 mL/min/1.73 m2). In ILLUMINATE-A, the percent reduction in 24-hour urinary oxalate from baseline to month 6 was -65% and -12% in the lumasiran and placebo group, respectively, with a between-group mean difference of 53% (95% CI: 45 to 62%; p<0.0001). A similar effect was seen in patients with high baseline urinary oxalate values, and approximately half of patients receiving lumasiran achieved normal urinary oxalate values by month 6. In ILLUMINATE-B, lumasiran demonstrated a percent reduction in spot urinary oxalate-to creatinine ratio from baseline of -71% (95% CI -77 to -65). The magnitude of the reduction and the time course were consistent with findings in ILLUMINATE-A. The major limitation is the lack of data on clinical outcomes such as nephrolithiasis (renal stones), nephrocalcinosis, and renal failure as both trials were not powered to assess these clinical endpoints. However, use of urinary oxalate as a surrogate for clinical outcomes in the pivotal trials may be justified based on the knowledge of the pathophysiology of the disease and the causal role of urinary oxalate in kidney stone formation, nephrocalcinosis, and loss of kidney function. Further, the consistency and size of treatment effect (more than half of patients receiving lumasiran achieved normal urinary oxalate levels at 6 months of treatment in ILLUMINATE-A) in clinical trials are indicative of the potential for a clinical benefit over the long term. Lumasiran was generally well-tolerated in ILLUMINATE-A and -B. However, the safety database was small and limited in duration. The evidence is sufficient to determine that the technology results in an improvement in the net health outcome.
 
2022 Update
Annual policy review completed with a literature search using the MEDLINE database through June 2022. No new literature was identified that would prompt a change in the coverage statement.

CPT/HCPCS:
C9074Injection, lumasiran, 0.5 mg
J0224Injection, lumasiran, 0.5 mg

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Group specific policy will supersede this policy when applicable. This policy does not apply to the Wal-Mart Associates Group Health Plan participants or to the Tyson Group Health Plan participants.
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