Subject: sapropterin-Javygtor-Kuvan 2012-A SGM P2024

Policy:

      I.  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.

          A.  FDA-Approved Indication
               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.

          B.  Compendial Uses
               1.  Autosomal dominant guanine triphosphate (GTP) cyclohydrolase deficiency (Segawa
                    disease)
               2.  Autosomal recessive guanine triphosphate (GTP) cyclohydrolase deficiency
               3.  6-pyruvoyl-tetrahydropterin synthase (6-PTS) deficiency
               4.  Sepiapterin reductase deficiency
               5.  Dihydropteridine reductase (DHPR) deficiency
               6.  Pterin-4a-carbinolamine dehydratase deficiency (also called primapterinuria)

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


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


   III.  CRITERIA FOR INITIAL APPROVAL

          A.  Phenylketonuria (PKU)
               Authorization of 60 days may be granted for members with a diagnosis of phenylketonuria
               who have a baseline phenylalanine level greater than or equal to 360 micromol/L (6 mg/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.

          B.  Biopterin Metabolic Defects
               Authorization of 6 months may be granted for members who have any of the following
               biopterin metabolic defects:
               1.  Autosomal dominant guanine triphosphate (GTP) cyclohydrolase deficiency (Segawa
                    disease)
               2.  Autosomal recessive guanine triphosphate (GTP) cyclohydrolase deficiency
               3.  6-pyruvoyl-tetrahydropterin synthase (6-PTS) deficiency
               4.  Sepiapterin reductase deficiency
               5.  Dihydropteridine reductase (DHPR) deficiency
               6.  Pterin-4a-carbinolamine dehydratase deficiency (also called primapterinuria)


   IV.  CONTINUATION OF THERAPY

          A.  Phenylketonuria (PKU)
               Authorization of 6 months may be granted for continued treatment in members requesting
               reauthorization for phenylketonuria (PKU) who meet any of the following criteria:
               1.  Achieve or maintain a 30% decrease in phenylalanine levels from baseline; or
               2.  Phenylalanine levels are in an acceptable range (less than 360 micromol/L or 6 mg/dL);
                    or
               3.  Demonstrate an improvement in neuropsychiatric symptoms.

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

          B.  Biopterin Metabolic Defects
               Authorization of 6 months may be granted for continued treatment in members requesting
               reauthorization for any biopterin metabolic defect listed in Section III 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.; February 2021.
2. Vockley J, Andersson HC, Antshel KM, et al. Phenylalanine hydroxylase deficiency: diagnosis and management guideline. Genet Med. 2014;16(2):188-200.
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.; May 2022.

 

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.

June 16, 2024