Subject: Empaveli 4738-A SGM P2022
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
Empaveli is indicated for the treatment of adult patients with
paroxysmal nocturnal hemoglobinuria (PNH).
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 for new requests for treatment
of:
A. For initial requests: flow cytometry used to show results of
glycosylphosphatidylinositol-anchored proteins (GPI-APs)
deficiency.
B. For continuation requests: Chart notes or medical record
documentation supporting positive clinical response.
III. CRITERIA FOR INITIAL APPROVAL
Paroxysmal nocturnal hemoglobinuria
Authorization of 6 months may be granted for treatment of
paroxysmal nocturnal hemoglobinuria (PNH) when all of the
following criteria are met:
A. The diagnosis of PNH was confirmed by detecting a
deficiency of glycosylphosphatidylinositol-anchored proteins
(GPI-APs) as demonstrated by either of the following:
1. At least 5% PNH cells
2. At least 51% of GPI-AP deficient poly-morphonuclear
cells
B. Flow cytometry is used to demonstrate GPI-APs deficiency
IV. CONTINUATION OF THERAPY
Paroxysmal nocturnal hemoglobinuria
Authorization of 12 months may be granted for continued
treatment in members requesting reauthorization when there is
no evidence of unacceptable toxicity or disease progression
while on the current regimen and demonstrate a positive
response to therapy (e.g., improvement in hemoglobin levels,
normalization of lactate dehydrogenase [LDH] levels).
V. DOSAGE AND ADMINISTRATION
Approvals may be subject to dosing limits in accordance with
FDA-approved labeling, accepted compendia, and/or evidence-
based practice guidelines.
Place of Service:
Outpatient
The above policy is based on the following references:
- Empaveli [package insert]. Waltham, MA: Apellis Pharmaceuticals, Inc.; May 2021
- Parker CJ. Management of paroxysmal nocturnal hemoglobinuria in the era of complement inhibitory therapy. 2011; 21-29.
- Borowitz MJ, Craig F, DiGiuseppe JA, et al. Guidelines for the Diagnosis and Monitoring of Paroxysmal Nocturnal Hemoglobinuria and Related Disorders by Flow Cytometry. Cytometry B Clin Cytom. 2010: 78: 211-230.
- Preis M, Lowrey CH. Laboratory tests for paroxysmal nocturnal hemoglobinuria (PNH). Am J Hematol. 2014;89(3):339-341.
- Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2016;2016(1):208-216.
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.
December 29, 2022
