Viltolarsen (Viltepso®)
IMPORTANT REMINDER
We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.
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
Viltepso is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with Viltepso. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
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. Initial requests:
1. Laboratory confirmation of Duchenne muscular dystrophy (DMD) diagnosis with a DMD gene mutation that is amenable to exon 53 skipping (refer to examples in Appendix).
2. If applicable, medical records confirming a worsening in clinical status since receiving gene replacement therapy.
B. Continuation of therapy requests: documentation (e.g., chart notes) of response to therapy.
III. PRESCRIBER SPECIALTIES
This medication must be prescribed by or in consultation with a physician who specializes in the treatment of Duchenne muscular dystrophy (DMD).
IV. CRITERIA FOR INITIAL APPROVAL
Duchenne Muscular Dystrophy
Authorization of 6 months may be granted for treatment of DMD when all of the following criteria are met:
A. Genetic testing was conducted to confirm the diagnosis of DMD and to identify the specific type of DMD gene mutation.
B. The DMD gene mutation is amenable to exon 53 skipping (refer to examples in Appendix).
C. Treatment with Viltepso is initiated before the age of 10.
D. Member is able to walk independently without assistive devices.
E. Member meets one of the following criteria:
1. Member has not previously received gene replacement therapy for DMD (e.g., Elevidys).
2. Member has previously received gene replacement therapy for DMD (e.g., Elevidys) and has experienced a worsening in clinical status since receiving gene replacement therapy (e.g., decline in ambulatory function).
F. Member will not exceed a dose of 80 mg/kg once weekly.
G. The requested medication will not be used concomitantly with golodirsen.
V. CONTINUATION OF THERAPY
Note: Members who were previously established on Viltepso and subsequently administered gene replacement therapy (e.g., Elevidys) must meet all initial criteria prior to re-starting Viltepso.
Authorization of 12 months may be granted for members requesting continuation of therapy when all of the following criteria are met:
A. The member has demonstrated a response to therapy as evidenced by remaining ambulatory (e.g., not wheelchair dependent).
B. The member will not exceed a dose of 80 mg/kg once weekly.
C. The requested medication will not be used concomitantly with golodirsen.
VI. APPENDIX
Examples of DMD gene mutations (exon deletions) amenable to exon 53 skipping (not an all-inclusive list):
1. Deletion of exon 52
2. Deletion of exon 45-52
3. Deletion of exon 47-52
4. Deletion of exon 48-52
5. Deletion of exon 49-52
6. Deletion of exon 50-52
APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS
BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.
ADDITIONAL INFORMATION
For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).
REFERENCES
1. Viltepso [package insert]. Paramus, NJ: NS Pharma, Inc.; March 2021.
2. Watanabe N, Nagata T, Satou Y, et al. NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy. Mol Ther Nucleic Acids. 2018;13:442–449. doi:10.1016/j.omtn.2018.09.017
3. Clinical Consult: CVS Caremark Clinical Programs Review. Focus on Neuromuscular Disorders Clinical Programs. September 2020.
ORIGINAL EFFECTIVE DATE: 10/31/2020
MOST RECENT REVIEW DATE: 1/1/2024
ID_CHS
Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.
This document has been classified as public information.