Subject: Kuvan 2012-A SGM P2021

Drug

KUVAN®  (sapropterin dihyrochloride)

sapropterin dihydrochloride (generic)

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 dehydralase 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 dehydralase 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.

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.

May 18, 2021