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
Exondys 51 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 51 skipping.
This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with Exondys 51. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.
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 51 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 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 51 skipping (refer to examples in Appendix).
C. Treatment with Exondys 51 is initiated before the age of 14.
D. Member is able to achieve an average distance of at least 180 meters while walking independently over 6 minutes.
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 30 mg/kg once weekly.
V. CONTINUATION OF THERAPY
Note: Members who were previously established on Exondys 51 and subsequently administered gene replacement therapy (e.g., Elevidys) must meet all initial criteria prior to re-starting Exondys 51.
Authorization of 12 months may be granted for members requesting continuation of therapy when both of the following criteria are met:
A. The member has demonstrated a response to therapy as evidenced by remaining ambulatory (e.g., able to walk with or without assistance, not wheelchair dependent).
B. The member will not exceed a dose of 30 mg/kg once weekly.
VI. APPENDIX
Examples of DMD gene mutations (exon deletions) amenable to exon 51 skipping (not an all-inclusive list):
1. Deletion of exon 50
2. Deletion of exon 52
3. Deletion of exons 45-50
4. Deletion of exons 47-50
5. Deletion of exons 48-50
6. Deletion of exons 49-50
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. Exondys 51 [package insert]. Cambridge, MA: Sarepta Therapeutics, Inc.; January 2022.
2. Mendell JR, Rodino-Klapac LR, Sahenk Z, et al. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol. 2013;74(5):637-47.
3. Cirak S, Arechavala-Gomeza V, Guglieri M, et al. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. Lancet. 2011;378(9791):595-605.
4. Mendell JR, Goemans N, Lowes LP, et al; Eteplirsen Study Group and Telethon Foundation DMD Italian Network. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann Neurol. 2016;79(2):257-271.
5. Randeree L, Eslick GD. Eteplirsen for paediatric patients with Duchenne muscular dystrophy: A pooled-analysis. J Clin Neurosci. 2018;49:1-6.
ORIGINAL EFFECTIVE DATE: 11/24/2017
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.