Subject: Voxzogo 5069-A SGM P2023a
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
Voxzogo is indicated to increase linear growth in pediatric
patients with achondroplasia with open epiphyses. This
indication is approved under accelerated approval based on an
improvement in annualized growth velocity. Continued approval
for this indication may be contingent upon verification and
description of clinical benefit in confirmatory trial(s).
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:
A. Chart notes or documentation of symptoms (i.e., short
stature with marked shortening of extremities due to
rhizomelia, a characteristic facial configuration, trident hand)
AND X-ray findings consistent with achondroplasia; OR
laboratory test reports of genetic testing for FGFR3 mutation
B. Growth chart
C. For continuation requests: Chart notes or medical record
documentation confirming benefit from therapy (e.g., growth
chart showing improvement or stabilization of annualized
growth velocity [centimeters per year])
III. PRESCRIBER SPECIALTIES
This medication must be prescribed by or in consultation with an
endocrinologist, pediatric endocrinologist, geneticist, or
neurologist.
IV. CRITERIA FOR INITIAL APPROVAL
Authorization of 12 months may be granted for treatment of
achondroplasia when ALL of the following criteria are met:
A. The diagnosis of achondroplasia was confirmed by EITHER of
the following:
1. Symptoms (i.e., short stature with marked shortening of
extremities due to rhizomelia, a characteristic facial
configuration, trident hand) AND X-ray findings consistent
with achondroplasia
2. Genetic testing for FGFR3 mutation
B. Epiphyses are open
V. CONTINUATION OF THERAPY
Authorization of 12 months may be granted for continuation of
therapy when all of the following criteria are met:
A. All criteria for initial approval are met
B. The member is experiencing benefit from therapy (e.g.,
improvement or stabilization of annualized growth velocity
[centimeters per year] from baseline)
Place of Service:
Outpatient
The above policy is based on the following references:
- Voxzogo [package insert]. Novato, CA: BioMarin Pharmaceutical Inc.; October 2023.
- Kubota T, Adachi M, Kitaoka T, et al. Clinical Practice Guidelines for Achondroplasia. Clin Pediatr Endocrinol. 2020;29(1):25-42.
- Trotter TL, Hall JG, American Academy of Pediatrics Committee on Genetics. Health supervision for children with achondroplasia. Pediatrics. 2005;116(3):771–783.
- Hoover-Fong J, Scott CI, Jones MC, Committee on Genetics. Health supervision for people with achondroplasia. Pediatrics. 2020;145(6):e20201010.
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 09, 2023
