Subject: sapropterin-Javygtor-Kuvan SGM 2012-A P2025

Drug

KUVAN  (sapropterin dihydrochloride)

JAVYGTOR  (sapropterin dihydrochloride)

Policy:

Indications

The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.

FDA-approved Indications^1,4,5

Kuvan/Javygtor/sapropterin is indicated to reduce blood phenylalanine (Phe) levels in adult and pediatric patients one month of age and older with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin-(BH4-) responsive phenylketonuria (PKU). Kuvan/Javygtor/saproprterin is to be used in conjunction with a Phe-restricted diet.

Compendial Uses

• Autosomal dominant guanine triphosphate (GTP) cyclohydrolase deficiency (Segawa disease)
• Autosomal recessive guanine triphosphate (GTP) cyclohydrolase deficiency
• 6-pyruvoyl-tetrahydropterin synthase (6-PTS) deficiency
• Sepiapterin reductase deficiency
• Dihydropteridine reductase (DHPR) deficiency
• Pterin-4a-carbinolamine dehydratase deficiency (also called primapterinuria)

All other indications are considered experimental/investigational and not medically necessary.

Documentation

Submission of the following information is necessary to initiate the prior authorization review: enzyme assay, genetic testing, or phenylalanine level results supporting diagnosis.

Prescriber Specialties

This medication must be prescribed by or in consultation with a physician who specializes in the treatment of metabolic disease and/or phenylketonuria (PKU).

Coverage Criteria

Phenylketonuria (PKU)^1-5

Authorization of 60 days may be granted when all of the following criteria are met:

• Member is one month of age or older.
• Member has been diagnosed with phenylketonuria and has a baseline phenylalanine level greater than or equal to 360 micromol/L (6mg/dL) with dietary interventions alone.

Note: If Kuvan is initiated in a member currently receiving Palynziq for phenylketonuria (PKU), then Palynziq will be discontinued after an appropriate period of overlap.

Biopterin Metabolic Defects

Authorization of 6 months may be granted for members one month of age and older who have any of the following biopterin metabolic defects:

• Autosomal dominant guanine triphosphate (GTP) cyclohydrolase deficiency (Segawa disease)
• Autosomal recessive guanine triphosphate (GTP) cyclohydrolase deficiency
• 6-pyruvoyl-tetrahydropterin synthase (6-PTS) deficiency
• Sepiapterin reductase deficiency
• Dihydropteridine reductase (DHPR) deficiency
• Pterin-4a-carbinolamine dehydratase deficiency (also called primapterinuria)

Continuation of Therapy

Phenylketonuria (PKU)^1,2,4,5

Authorization of 6 months may be granted for continued treatment in members requesting reauthorization for phenylketonuria (PKU) who meet any of the following criteria:

• Achieve or maintain a 30% decrease in phenylalanine levels from baseline; or
• Phenylalanine levels are in an acceptable range (less than 360 micromol/L or 6mg/dL); or
• Demonstrate an improvement in neuropsychiatric symptoms.

Note: Kuvan should not be used concomitantly with Palynziq for phenylketonuria (PKU).

Biopterin Metabolic Defects

Authorization of 6 months may be granted for continued treatment in members requesting reauthorization for any biopterin metabolic defect listed in the coverage criteria section who are experiencing benefit from therapy as evidenced by disease stability or disease improvement.

Place of Service:

Outpatient

The above policy is based on the following references:

1. Kuvan [package insert]. Novato, CA: BioMarin Pharmaceutical Inc.; August 2024.
2. Smith WE, Berry SA, Bloom K, et al. Phenylalanine hydroxylase deficiency diagnosis and management: A 2023 evidence-based clinical guideline of the American College of Medical Genetics and Genomics (ACMG). Genet Med. Published online December 2, 2024. doi:10.1016/j.gim.2024.101289
3. Singh RH, Rohr F, Frazier D, et al. Recommendations for the nutrition management of phenylalanine hydroxylase deficiency. Genet Med. 2014;16(2):121-131.
4. Sapropterin dihydrochloride [package insert]. Chestnut Ridge, NY: Par Pharmaceutical; April 2020.
5. Javygtor [package insert]. Princeton, NJ: Dr. Reddy’s Laboratories, Inc.; October 2024.

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

July 22, 2025