Subject: Zokinvy 4384-A SGM P2024
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 Indications
Zokinvy is indicated in patients 12 months of age and older with a body surface area (BSA) of
0.39 m2 and above:
A. To reduce risk of mortality in Hutchinson-Gilford Progeria Syndrome (HGPS)
B. For the treatment of processing-deficient Progeroid Laminopathies with heterozygous
LMNA mutation with progerin-like protein accumulation
C. For the treatment of processing-deficient Progeroid Laminopathies with homozygous or
compound heterozygous ZMPSTE24 mutations
Limitations of Use
Zokinvy is not indicated for other Progeroid Syndromes or processing-proficient Progeroid
Laminopathies. Based upon its mechanism of action, Zokinvy would not be expected to be
effective in these populations.
All other indications are considered experimental/investigational and not medically necessary.
II. DOCUMENTATION
A. Hutchinson-Gilford Progeria Syndrome
Submission of the following information is necessary to initiate the prior authorization review
for Hutchinson-Gilford Progeria Syndrome: Genetic testing results confirming the member
has a LMNA mutation.
B. Processing-Deficient Progeroid Laminopathy with Progerin-Like Protein
Accumulation
Submission of the following information is necessary to initiate the prior authorization review
for Processing-Deficient Progeroid Laminopathy with Progerin-Like Protein Accumulation:
Genetic testing results confirming the member has a heterozygous LMNA mutation.
C. Processing-Deficient Progeroid Laminopathy without Progerin-Like Protein
Accumulation
Submission of the following information is necessary to initiate the prior authorization review
for Processing-Deficient Progeroid Laminopathy without Progerin-Like Protein
Accumulation: Genetic testing results confirming the member has either homozygous or
compound heterozygous ZMPSTE24 mutations.
III. CRITERIA FOR INITIAL APPROVAL
A. Hutchinson-Gilford Progeria Syndrome
Authorization of 12 months may be granted for treatment of Hutchinson-Gilford Progeria
Syndrome when all of the following criteria are met:
1. The member is 12 months of age or older.
2. The member has a body surface area of 0.39 m2 or above.
3. The diagnosis of Hutchinson-Gilford Progeria Syndrome has been confirmed with
genetic testing indicating the member has a LMNA
B. Processing-Deficient Progeroid Laminopathy with Progerin-Like Protein
Accumulation
Authorization of 12 months may be granted for treatment of Processing-Deficient Progeroid
Laminopathy with Progerin-Like Protein Accumulation when all of the following criteria are
met:
1. The member is 12 months of age or older.
2. The member has a body surface area of 0.39 m2 or above.
3. The diagnosis of Processing-Deficient Progeroid Laminopathy has been confirmed with
genetic testing indicating the member has a heterozygous LMNA
C. Processing-Deficient Progeroid Laminopathy without Progerin-Like Protein
Accumulation
Authorization of 12 months may be granted for treatment of Processing-Deficient Progeroid
Laminopathy without Progerin-Like Protein Accumulation when all of the following criteria
are met:
1. The member is 12 months of age or older.
2. The member has a body surface area of 0.39 m2 or above.
3. The diagnosis of Processing-Deficient Progeroid Laminopathy has been confirmed with
genetic testing indicating the member has homozygous or compound heterozygous
ZMPSTE24
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 when all of the following criteria are met:
A. Member meets all initial authorization criteria.
B. Member is experiencing benefit from therapy.
Place of Service:
Outpatient
The above policy is based on the following references:
- Zokinvy [package insert]. Palo Alto, CA: Eiger BioPharmaceuticals, Inc.; November 2020.
- Progeria Research Foundation (PRF). The Progeria Handbook: A Guide for Families & Health Care Providers of Children with Progeria. Second Edition. PRF. https://www.progeriaresearch.org/wp-content/uploads/2019/03/PRF_Handbook_2019_eFile.pdf. Accessed November 27, 2020.
- Gordon LB, Brown WT, Collins FS. Hutchinson-Gilford Progeria Syndrome. 2003 Dec 12 [Updated 2023 Oct 19]. In: Adam MP, Feldman J, Mirzaa GM, et al., editors. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2024. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1121/
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.
August 18, 2024
