Subject: sapropterin-Javygtor-Kuvan 2012-A SGM P2023

Drug

KUVAN  (sapropterin dihydrochloride)

JAQYGTOR  (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/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
               (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 (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 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.; 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 18, 2023