Pharmacy Prior Authorization Criteria

Exondys 51 (eteplirsen) - PA, NF

Indications for Prior Authorization

Exondys 51 (eteplirsen)

• 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 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.

Criteria

Exondys 51

Prior Authorization (Initial Authorization)

Length of Approval: 6 Month(s)

• Diagnosis of Duchenne muscular dystrophy (DMD)
AND
• Patient has a mutation of the dystrophin gene amenable to exon 51 skipping
AND
• Patient is 7 years of age or older [2-4]
AND
• Prescribed by or in consultation with a neurologist who has experience treating children
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.) [2-4]

Exondys 51

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

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

Exondys 51

Non Formulary

Length of Approval: 6 Month(s)

• Submission of medical records (e.g., chart notes, laboratory values) confirming both of the following:
• Diagnosis of Duchenne muscular dystrophy (DMD)
AND
• Patient has a mutation of the dystrophin gene amenable to exon 51 skipping
AND
• Patient is 7 years of age or older [2-4]
AND
• Prescribed by or in consultation with a neurologist who has experience treating children
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) confirming the patient is ambulatory without needing an assistive device (e.g., without side-by-side assist, cane, walker, wheelchair, etc.) [2-4]

P & T Revisions

2024-05-03, 2023-12-11, 2023-10-26, 2023-05-04, 2022-06-02, 2022-05-05, 2021-12-16, 2021-06-02, 2021-05-04, 2020-04-14, 2020-01-29

References

1. Exondys 51 Prescribing Information. Sarepta Therapeutics, Inc. Cambridge, MA. January 2022.
2. Mendell JR, Goemans N, Lowes LP, et al. Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy. Ann Neurol. 2016;79(2):257-271. doi: 10.1002/ana.24555
3. Mendell JR, Rodino-Klapac LR, Sahenk Z, et al. Eteplirsen for the treatment of Duchenne muscular dystrophy. Ann Neurol. 2013;74(5):637-647.
4. Per Clinical Consultation with a Pediatrician, October 5, 2016 and January 22, 2020.

---

Revision History

• 2024-05-03: Annual review: Updated criteria and background.
• 2023-12-11: Update to reauth language
• 2023-10-26: Program Update
• 2023-05-04: Annual review: Formatting updates.
• 2022-06-02: Removed submission of medical records and/or paid claims requirement from prior authorization criteria.
• 2022-05-05: Annual review: Background updates.
• 2021-12-16: Addition of NF criteria
• 2021-06-02: Updated reauthorization criteria.
• 2021-05-04: 2021 Annual Review, updated indication to align with labeling. No changes to clinical criteria.
• 2020-04-14: 2020 Annual Review, no changes to criteria
• 2020-01-29: Updated initial and reauthorization criteria and background.

---