Pharmacy Prior Authorization Criteria

Vyondys 53 (golodirsen) - PA, NF

Indications for Prior Authorization

Vyondys 53 (golodirsen)

• For diagnosis of Duchenne muscular dystrophy (DMD)
  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 VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

Criteria

Vyondys 53

Prior Authorization (Initial Authorization)

Length of Approval: 6 Month(s)

• Diagnosis of Duchenne muscular dystrophy (DMD)
AND
• Documentation of a confirmed mutation of the dystrophin gene amenable to exon 53 skipping
AND
• Patient is 6 years of age or older [2, 3]
AND
• Prescribed by or in consultation with a neurologist with expertise in the treatment of DMD
AND
• Dose will not exceed 30 milligrams per kilogram of body weight infused once weekly
AND
• Patient is ambulatory without needing an assistive device (e.g., without side-by-side assist, cane, walker, wheelchair, etc.) [2, 3]

Vyondys 53

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

• Patient is tolerating therapy
AND
• Dose will not exceed 30 milligrams per kilogram of body weight infused once weekly
AND
• Prescribed by or in consultation with a neurologist with expertise in the treatment of DMD
AND
• Patient is maintaining ambulatory status without needing an assistive device (e.g., without side-by-side assist, cane, walker, wheelchair, etc.)

Vyondys 53

Non Formulary

Length of Approval: 6 Month(s)

• Submission of medical records (e.g., chart notes, laboratory values) documenting both of the following:
• Diagnosis of Duchenne muscular dystrophy (DMD)
AND
• Confirmed mutation of the dystrophin gene amenable to exon 53 skipping
AND
• Patient is 6 years of age or older [2, 3]
AND
• Prescribed by or in consultation with a neurologist with expertise in the treatment of DMD
AND
• Dose will not exceed 30 milligrams per kilogram of body weight infused once weekly
AND
• Submission of medical records (e.g., chart notes, laboratory values) documenting the patient is ambulatory without needing an assistive device (e.g., without side-by-side assist, cane, walker, wheelchair, etc.) [2, 3]

P & T Revisions

2025-01-29, 2024-01-31, 2023-12-11, 2023-10-26, 2023-02-02, 2022-06-02, 2022-02-03, 2021-12-16, 2021-06-02, 2021-02-03, 2020-01-29, 2019-12-17

References

1. Vyondys 53 Prescribing Information. Sarepta Therapeutics, Inc. Cambridge, MA. June 2024.
2. Muntoni F, Frank DE, Morgan J, et al. Golodirsen induces exon skipping leading to sarcolemmal dystrophin expression in patients with genetic mutations amenable to exon 53 skipping [abstract]. Neuromuscul Disord. 2018;28:S5. Abstract D01.
3. Per Clinical Consultation with a Pediatrician, April 25, 2019 and January 22, 2020.

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Revision History

• 2025-01-29: 2025 Annual Review. Updated prescriber requirement. Updated references.
• 2024-01-31: Annual review: Updated non formulary criteria.
• 2023-12-11: Update to reauth language.
• 2023-10-26: Program Update
• 2023-02-02: Annual review: No updates required.
• 2022-06-02: Removed submission of medical records and/or paid claims requirement from prior authorization criteria.
• 2022-02-03: Annual review: No updates required.
• 2021-12-16: Addition of NF criteria
• 2021-06-02: Updated reauthorization criteria.
• 2021-02-03: Annual review: Background updates.
• 2020-01-29: Updated initial and reauthorization criteria and background.
• 2019-12-17: New Program.

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