60923-0363-XX EXONDYS 51 100MG/2ML Solution (SAREPTA THERAPEUTICS)
60923-0284-XX EXONDYS 51 500MG/10ML Solution (SAREPTA THERAPEUTICS)
Eteplirsen is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy. Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in individuals with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. Eteplirsen treated individuals were found to produce messenger ribonucleic acid (mRNA) for a truncated dystrophin protein by reverse transcription polymerase chain reaction.
Eteplirsen for the treatment of Duchenne muscular dystrophy is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Eteplirsen for the treatment of other conditions/diseases is considered investigational.
Eteplirsen is considered medically appropriate if ALL of the following criteria are met:
Individual is not on concomitant therapy with other DMD-directed antisense oligonucleotides (e.g., eteplirsen, etc.)
Confirmed mutation of the Duchenne muscular dystrophy (DMD) gene that is amenable to exon 51 skipping
The individual has been on a stable dose of corticosteroids, unless contraindicated or intolerance, for at least 6 months
The individual retains meaningful voluntary motor function (e.g., able to speak, manipulate objects using upper extremities, ambulate)
The individual is receiving physical and/or occupational therapy
Baseline documentation of ANY ONE or more of the following:
6-minute walk test (6MWT) or other timed function tests
Upper limb function (ULM) test
North Star Ambulatory Assessment (NSAA)
Forced Vital Capacity (FVC) percent predicted
Eteplirsen is considered medically appropriate for renewal if ALL of the following criteria are met:
Individual continues to meet the initial approval criteria (not including prerequisite therapy)
Absence of unacceptable toxicity from the agent (e.g., severe hypersensitivity reactions, balance disorders, vomiting, etc.)
Individual has received benefit from therapy, which may include ANY ONE or more of the following:
Increase in dystrophin
Stability, improvement, or slowed rate of decline in 6MWT or other timed function tests
Stability, improvement, or slowed rate of decline in ULM test
Stability, improvement, or slowed rate of decline in NSAA
Stability, improvement, or slowed rate of decline in FVC% predicted
Improvement in quality of life
DOSAGE & ADMINISTRATION
Duchenne muscular dystrophy
30 mg / kg administered once weekly as a 35 to 60 minute intravenous infusion
LENGTH OF AUTHORIZATION
Coverage will be provided for 6 months and may be renewed.
Refer to DOSAGE LIMITS below
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.
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, the express terms of the health plan will govern.
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).
Khan, N., Eliopoulos, H., Han, L., Kinane, T. B., Lowes, L. P., Mendell, J. R., et al. (2019). Eteplirsen treatment attenuates respiratory decline in ambulatory and non-ambulatory patients with Duchenne muscular dystrophy. Journal of Neuromuscular Disease, 6(2019). 213-215.
Lexicomp Online. (2020). AHFS DI. Eteplirsen. Retrieved February 13, 2020 from Lexicomp Online with AHFS.
Mercuri, E., Coratti, G., Messina, S., Ricotti, V., Baranello, G., D’Amico, A., et al. (2016). Revised North Star Ambulatory Assessment for young boys with Duchenne muscular dystrophy. PLoS ONE, 11(8). 1-9.
MICROMEDEX Healthcare Series. Drugdex Evaluations. (2019, November). Eteplirsen. Retrieved February 13, 2020 from MICROMEDEX Healthcare Series.
U. S. Food and Drug Administration. (2018, October). Center for Drug Evaluation and Research. Exondys 51™ (eteplirsen). Retrieved February 13, 2020 from https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/206488s009lbl.pdf.
ORIGINAL EFFECTIVE DATE: 11/24/2017
MOST RECENT REVIEW DATE: 6/30/2020
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.
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Maximum billable units per dose and over time by indication as a Medical Benefit; 1 billable unit = 10 mg