Subject: sapropterin-Kuvan 2012-A SGM P2022

Drug

KUVAN  (sapropterin dihydrochloride)

sapropterin dihydrochloride

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 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 is to be used in
                 conjunction with a Phe-restricted diet.

            B.  Compendial Uses
                 1.  Autosomal dominant guanine triphosphate
                      cyclohydrolase deficiency (Segawa disease)
                 2.  Autosomal recessive guanine (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 (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.

           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
                     cyclohydrolase deficiency (Segawa disease)
                2.  Autosomal recessive guanine (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 6mg/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.; March 2020.
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.

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 26, 2022