Subject: nitisinone-Nityr-Orfadin 2120-A SGM P2024

Drug

NITYR  (nitisinone)

ORFADIN  (nitisinone)

nitisinone

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.

          FDA-Approved Indication
          Nityr/Orfadin/nitisinone is indicated for the treatment of adult and pediatric patients with
          hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and
          phenylalanine.

          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:
          biochemical testing, enzyme assay, or genetic testing results supporting diagnosis.


   III.  CRITERIA FOR INITIAL APPROVAL

          Hereditary Tyrosinemia Type 1 (HT-1)
          Authorization of 12 months may be granted for treatment of hereditary tyrosinemia type 1 (HT-
          1) when the diagnosis is confirmed by biochemical testing (e.g., detection of succinylacetone in
          urine), enzyme assay, or genetic testing and the requested medication is being used as an
          adjunct to dietary restriction of tyrosine and phenylalanine.


   IV.  CONTINUATION OF THERAPY

          Authorization of 12 months may be granted for continued treatment in members requesting
          reauthorization for an indication listed in Section III who are experiencing beneficial clinical
          response from therapy.

 

Place of Service:

Outpatient

The above policy is based on the following references:

1. Orfadin [package insert]. Waltham, MA: Sobi, Inc; November 2021.
2. Nityr [package insert]. Cambridge, United Kingdom: Cycle Pharmaceuticals Ltd.; June 2021.
3. nitisinone [package insert]. Chestnut Ridge, NY: Par Pharmaceutical; October 2019.

 

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.

April 17, 2024