Subject: nitisinone-Nityr-Orfadin 2120-A SGM P2023

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
          Orfadin 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.

          Nityr 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

          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) or DNA 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 hereditary
          tyrosinemia type 1 (HT-1) 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.

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