Subject: Voxzogo SGM 5069-A P2025

Drug

VOXZOGO  (vosoritide)

Policy:

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 Indications¹

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.

Documentation

Submission of the following information is necessary to initiate the prior authorization review:

• Chart notes or documentation of symptoms (i.e., short stature with marked shortening of extremities due to rhizomelia, a characteristic facial configuration, trident hand) (if applicable)
• Laboratory report of X-ray findings consistent with achondroplasia or laboratory report of genetic testing for FGFR3 mutation
• Growth chart
• 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])

Prescriber Specialties

This medication must be prescribed by or in consultation with an endocrinologist, pediatric endocrinologist, geneticist, or neurologist.

Coverage Criteria

Achondroplasia

Authorization of 12 months may be granted for treatment of achondroplasia when BOTH of the following criteria are met:

• The diagnosis of achondroplasia was confirmed by EITHER of the following:
  • 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
  • Genetic testing for FGFR3 mutation
• Epiphyses are open

Continuation of Therapy

Authorization of 12 months may be granted for continuation of therapy when BOTH of the following criteria are met:

• Member meets all requirements in the coverage criteria
• 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:

1. Voxzogo [package insert]. Novato, CA: BioMarin Pharmaceutical Inc.; November 2024.
2. Kubota T, Adachi M, Kitaoka T, et al. Clinical Practice Guidelines for Achondroplasia. Clin Pediatr Endocrinol. 2020;29(1):25-42.
3. Trotter TL, Hall JG, American Academy of Pediatrics Committee on Genetics. Health supervision for children with achondroplasia. Pediatrics. 2005;116(3):771–783.
4. 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.

June 07, 2025