Clinical Policy: Golodirsen (Vyondys 53)

Reference Number: CP.PHAR.453
Effective Date: 03.01.20
Last Review Date: 02.26
Line of Business: Commercial, HIM, Medicaid

[Coding Implications](Coding Implications)
[Revision Log](Revision Log)

See [Important Reminder](Important Reminder) at the end of this policy for important regulatory and legal information.

Description

Golodirsen (Vyondys 53®) is an antisense oligonucleotide.

FDA Approved Indication(s)

Vyondys 53 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.

Limitation(s) of use: This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with Vyondys 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

Policy/Criteria

7. If request is for a dose increase, new dose does not exceed 30 mg/kg per week.

Approval duration: 6 months

III. Diagnoses/Indications for which coverage is NOT authorized:

A. Non-FDA approved indications, which are not addressed in this policy, unless there is sufficient documentation of efficacy and safety according to the off label use policies – CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid, or evidence of coverage documents.

IV. Appendices/General Information

Appendix A: Abbreviation/Acronym Key

6MWT: 6-minute walk test
DMD: Duchenne muscular dystrophy
FDA: Food and Drug Administration
FVC: forced vital capacity
ICER: Institute for Clinical and Economic Review
LVEF: left ventricular ejection fraction

Appendix B: Therapeutic Alternatives

This table provides a listing of preferred alternative therapy recommended in the approval criteria. The drugs listed here may not be a formulary agent for all relevant lines of business and may require prior authorization.

Therapeutic alternatives are listed as Brand name® (generic) when the drug is available by brand name only and generic (Brand name®) when the drug is available by both brand and generic.
*Off-label

Appendix C: Contraindications/Boxed Warnings

• Contraindication(s): serious hypersensitivity to golodirsen or to any of the inactive ingredients in Vyondys 53
• Boxed warning(s): none reported

Appendix D: General Information

• Common mutations amenable to exon 53 skipping include: 3-52, 4-52, 5-52, 6-52, 9-52, 10-52, 11-52, 13-52, 14-52, 15-52, 16-52, 17-52, 19-52, 21-52, 23-52, 24-52, 25-52, 26-52, 27-52, 28-52, 29-52, 30-52, 31-52, 32-52, 33-52, 34-52, 35-52, 36-52, 37-52, 38-52, 39-52, 40-52, 41-52, 42-52, 43-52, 45-52, 47-52, 48-52, 49-52, 50-52, 52, 54-58, 54-61, 54-64, 54-66, 54-76, 54-77.
• Corticosteroids are routinely used in DMD management with established efficacy in slowing decline of muscle strength and function (including motor, respiratory, and cardiac). They are recommended for all DMD patients per the American Academy of Neurology (AAN) and DMD Care Considerations Working Group; in addition, the AAN guidelines have been endorsed by the American Academy of Pediatrics, the American Association of Neuromuscular & Electrodiagnostic Medicine, and the Child Neurology Society.
  
  • The DMD Care Considerations Working Group guidelines, which were updated in 2018, continue to recommend corticosteroids as the mainstay of therapy.
  • In an evidence report published August 2019, the Institute for Clinical and Economic Review (ICER) states that current evidence is insufficient to conclude that Vyondys 53 has net clinical benefit when added to corticosteroids and supportive care alone versus corticosteroids and supportive care alone.
• Prednisone is the corticosteroid with the most available evidence. A second corticosteroid commonly used is deflazacort, which was FDA approved for DMD in February 2017. On October 2023, a third corticosteroid, vamorolone, was approved by the FDA for DMD.
• The inclusion criteria for Study 4053-US-101 (NCT02310906) used to support the FDA approval of Vyondys 53 enrolled male patients age 6-15 years old with a mean 6MWT distance of 250 m or more at screening and baseline visits, LVEF ≥ 50% based on screening echocardiogram (ECHO), and stable pulmonary function with FVC ≥ 50%.

V. Dosage and Administration

VI. Product Availability

Single-dose vial for injection: 100 mg/2 mL (50 mg/mL)

Coding Implications

Codes referenced in this clinical policy are for informational purposes only. Inclusion or exclusion of any codes does not guarantee coverage. Providers should reference the most up-to-date sources of professional coding guidance prior to the submission of claims for reimbursement of covered services.

Reviews, Revisions, and Approvals

1Q 2022 annual review: no significant changes; clarified that LVEF “inclusive of 50% per pivotal study design; added that the review “may” require medical director review; references reviewed and updated.
1Q 2023 annual review: no significant changes; updated Section III to match template; references reviewed and updated.
1Q 2024 annual review: added criteria, member has not previously received gene replacement therapy for DMD (e.g., Elevidys); added Agamree to list of corticosteroids in Appendix B; references updated and reviewed.
1Q 2025 annual review: no significant changes; updated Appendix C with new contraindication per PI; references reviewed and updated.
1Q 2026 annual review: no significant changes; added step therapy bypass for IL HIM per IL HB 5395; references reviewed and updated.

Important Reminder

This clinical policy has been developed by appropriately experienced and licensed health care professionals based on a review and consideration of currently available generally accepted standards of medical practice; peer-reviewed medical literature; government agency/program approval status; evidence-based guidelines and positions of leading national health professional organizations; views of physicians practicing in relevant clinical areas affected by this clinical policy; and other available clinical information. The Health Plan makes no representations and accepts no liability with respect to the content of any external information used or relied upon in developing this clinical policy. This clinical policy is consistent with standards of medical practice current at the time that this clinical policy was approved. “Health Plan” means a health plan that has adopted this clinical policy and that is operated or administered, in whole or in part, by Centene Management Company, LLC, or any of such health plan’s affiliates, as applicable.