Subject: deferiprone-Ferriprox 1621-A SGM P2023

Drug

FERRIPROX  (deferiprone)

deferiprone

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 Indications
                1.  Transfusional Iron Overload due to Thalassemia
                      Syndromes
                      a.  Oral solution is indicated for treatment of 
                           transfusional iron overload in adult and pediatric
                           patients 3 years of age and older with thalassemia
                           syndromes.
                      b.  Tablets are indicated for treatment of transfusional
                           iron overload in adult and pediatric patients 8 years
                           of age and older with thalassemia syndromes.

                2.  Transfusional Iron Overload due to Sickle Cell
                     Disease or Other Anemias
                     a.  Ferriprox oral solution is indicated for the treatment
                          of transfusional iron overload in adult and pediatric
                          patients 3 years of age and older with sickle cell
                          disease or other anemias.
                     b.  Ferriprox tablets are indicated for treatment of
                          transfusional iron overload in adult and pediatric
                          patients 8 years of age and older with sickle cell
                          disease or other anemias.

          B.  Compendial Use
               Hereditary hemochromatosis
          
               Limitations of Use
               Safety and effectiveness have not been established for the
               treatment of transfusional iron overload in patients with
               myelodysplastic syndrome or in patients with Diamond
               Blackfan anemia.

          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:

          Transfusional Iron Overload:
          A.  Initial requests: pretreatment serum ferritin level
          B.  Continuation requests: current serum ferritin level

 
   III.  CRITERIA FOR INITIAL APPROVAL

           A.  Transfusional Iron Overload
                Authorization of 6 months may be granted for treatment of
                transfusional iron overload when all of the following criteria
                are met:
                1.  Transfusional iron overload is due to either of the
                     following:
                     a.  Thalassemia syndromes
                     b.  Sickle cell disease or other anemias
                2.  Member does not have transfusional iron overload due to
                     myelodysplastic syndrome or Diamond Blackfan anemia
                3.  Pretreatment serum ferritin level is consistently greater
                     than 1000 mcg/L.
                4.  Dose of Ferriprox will not exceed 99 mg/kg per day.

           B.  Hereditary Hemochromatosis
                Authorization of 6 months may be granted for treatment of
                hereditary hemochromatosis when phlebotomy is not an
                option (e.g., poor candidate due to underlying medical
                disorders) or the member had an unsatisfactory response to
                phlebotomy.

    IV.  CONTINUATION OF THERAPY

           A.  Transfusional Iron Overload
                Authorization of 6 months may be granted for continued
                treatment in members requesting reauthorization for
                transfusion iron overload when following criteria are met:
                1.  Member is experiencing benefit from therapy as
                     evidenced by a decrease in serum ferritin levels as
                     compared to pretreatment baseline.
                2.  Serum ferritin level is not consistently below 500 mcg/L.

           B.  Hereditary Hemochromatosis
                Authorization of 6 months may be granted for continued
                treatment in members requesting reauthorization for
                hereditary hemochromatosis when member is experiencing
                benefit from therapy as evidenced by a decrease in serum
                ferritin levels as compared to pretreatment baseline

Place of Service:

Outpatient

The above policy is based on the following references:

1. Ferriprox tablets [package insert]. Cary, NC: Chiesi USA, Inc.; November 2021.
2. Ferriprox oral solution [package insert]. Cary, NC: Chiesi USA, Inc.; November 2021.
3. Deferiprone [package insert]. Hawthorne, NY: Taro Pharmaceuticals USA., Inc.; August 2022
4. Deferiprone [package insert]. Berkeley Heights, NJ:Hikma Pharmaceuticals USA Inc.; December 2021
5. Cappellini MD, Cohen A, Porter J, et al. Guidelines for the management of transfusion dependent thalassaemia (TDT) 4^th Edition [Internet]. Thalassaemia International Federation 2021;20:1-351..
6. Hoffbrand AV, Taher A, Cappellini MD. How I treat transfusional iron overload. Blood 2012;120(18):3657-69
7. Kowdley, Kris V. MD, FACG1; Brown, Kyle E. MD, MSc2,3,4; Ahn, Joseph MD, MS, MBA, FACG (GRADE Methodologist)5; Sundaram, Vinay MD, MSc6 ACG Clinical Guideline: Hereditary Hemochromatosis, The American Journal of Gastroenterology: August 2019 - Volume 114 - Issue 8 - p 1202-1218

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 11, 2023