CurrentNeighborhood Health Plan of Rhode IslandPolicy 2012-A

Sapropterin (Kuvan/Javygtor) coverage for PKU and biopterin metabolic defects — prior authorization and coverage criteria

Defines prior authorization, clinical criteria, and coverage conditions for sapropterin (Kuvan/Javygtor) for members of Neighborhood Health Plan of Rhode Island, including FDA-approved use in BH4-responsive PKU and specified biopterin metabolic defects.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicySapropterin (Kuvan/Javygtor) coverage for PKU and biopterin metabolic defects

Policy CodePolicy 2012-A

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionSubmit prior authorization with supporting documentation (enzyme assay, genetic testing, or phenylalanine levels) to request coverage.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

1 monthminimum patient age for coverage

>=360baseline Phe threshold

30%required Phe decrease for continuation

6 monthsauthorization period (biopterin defects/reauth)

60 daysinitial PKU authorization period

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Coverage Criteria for Sapropterin (Kuvan / Javygtor)

Initial Therapy - PKU

Covered when ALL of the following are met:

Initial PKU authorization: Member is one month of age or older AND member has a diagnosis of phenylketonuria AND baseline phenylalanine (Phe) level greater than or equal to 360 micromol/L (6 mg/dL) despite dietary interventionsage >= 1 month; Phe >= 360 micromol/L (6 mg/dL)

Authorization of 60 days may be granted

Initial Therapy - Biopterin Defects

Covered when ALL of the following are met:

Biopterin metabolic defects authorization: Member is one month of age or older AND member has one of the listed biopterin metabolic defects: autosomal dominant GTP cyclohydrolase deficiency (Segawa disease), autosomal recessive GTP cyclohydrolase deficiency, 6-pyruvoyl-tetrahydropterin synthase (6-PTS) deficiency, sepiapterin reductase deficiency, dihydropteridine reductase (DHPR) deficiency, or pterin-4a-carbinolamine dehydratase deficiencyage >= 1 month

Authorization of 6 months may be granted

Continuation / Reauthorization

Covered when ANY of the following are met:

PKU reauthorization criteria: Member achieves or maintains a 30% decrease in phenylalanine levels from baseline OR phenylalanine levels are less than 360 micromol/L (6 mg/dL) OR member demonstrates improvement in neuropsychiatric symptoms>= 30% decrease OR Phe < 360 micromol/L (6 mg/dL)

Authorization of 6 months may be granted; Kuvan should not be used concomitantly with Palynziq for PKU

Biopterin defects reauthorization: Member with a listed biopterin metabolic defect is experiencing benefit from therapy as evidenced by disease stability or disease improvementclinical benefit demonstrated

Authorization of 6 months may be granted

All other indications not specifically listed in this policy are considered experimental/investigational and are not covered.

Indications other than the FDA‑approved use and the compendial uses specified in this policy are not medically necessary and will not be covered.

Clinical Thresholds and Coding-relevant Values

Phenylalanine (Phe) level — thresholds and response criteria

Baseline threshold for initial PKU authorizationPhenylalanine (Phe) >= 360 micromol/L (6 mg/dL)

Age requirement at baselineMember is one month of age or older

Continued-treatment response — Phe absolute levelPhenylalanine (Phe) < 360 micromol/L (6 mg/dL)

Continued-treatment response — relative changeAchieve or maintain a >= 30% decrease in Phe from baseline

Authorization durationsInitial PKU authorization: up to 60 days; reauthorization for continued treatment: up to 6 months

Prescriber Requirements, Prior Authorization, and Documentation

Prior Authorization

Prior authorization required; limited-duration approvals

Prior authorization is required. For initial PKU treatment, authorization of up to 60 days may be granted when the coverage criteria are met; for initial therapy and reauthorization for biopterin metabolic defects, and for PKU reauthorization when continued-treatment criteria are met, authorization of 6 months may be granted.

Step Therapy

Concomitant therapy restriction with Palynziq

Kuvan must not be used concomitantly with Palynziq for PKU. If Kuvan is initiated in a member currently receiving Palynziq, Palynziq will be discontinued after an appropriate period of overlap.

Documentation Required

Key Definitions

BH4‑responsive PKU — definition and policy response concept

DefinitionBH4-responsive phenylketonuria (PKU) is PKU due to phenylalanine hydroxylase deficiency that responds to tetrahydrobiopterin (BH4) supplementation, resulting in reduced blood phenylalanine (Phe) levels when treated with sapropterin (Kuvan/Javygtor).

Clinical response concept used by policyResponse is demonstrated by a clinically meaningful reduction in blood Phe — defined in this policy as either Phe < 360 micromol/L (6 mg/dL) or a >= 30% decrease from baseline — or by observable clinical improvement (e.g., neuropsychiatric symptoms).

Treatment contextSapropterin is indicated for BH4-responsive PKU in patients one month of age and older and is to be used with a Phe‑restricted diet.

Background

Sapropterin (Kuvan/Javygtor) is indicated to reduce blood phenylalanine (Phe) levels in patients one month of age and older with hyperphenylalaninemia due to BH4‑responsive phenylketonuria and is used in conjunction with a Phe‑restricted diet. It acts as a synthetic form of tetrahydrobiopterin (BH4) to enhance residual phenylalanine hydroxylase activity and lower circulating Phe; the agent is also used for certain biopterin metabolic defects listed in compendia.