VILTEPSO (viltolarsen)

VILTEPSO (viltolarsen)

OFFICE ADMINISTRATION - INTRAVENOUS

Indications for Prior Authorization:

Viltepso is an antisense oligonucleotide 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 Viltespo.

Patients must meet the following criteria:

Duchenne muscular dystrophy (DMD) (Initial authorization):

• Submission of medical records (e.g., chart notes, laboratory values) documenting both of the following:
  • Diagnosis of Duchenne muscular dystrophy (DMD); AND
  • Documentation of a confirmed mutation of the dystrophin gene amenable to exon 53 skipping; AND
• Patient is 4 years of age or older; AND
• Prescribed by or in consultation with a neurologist with expertise in the treatment of DMD; AND
• Dose does not exceed 80 mg/kg of body weight infused once weekly; AND
• Submission of medical records (e.g., chart notes, laboratory values) documenting the patient is ambulatory, as evaluated via the 6-minute walk test (6MWT) or North Star ambulatory assessment (NSAA).

Duchenne muscular dystrophy (DMD) (Reauthorization):

• Submission of medical records (e.g., chart notes, laboratory values) documenting both of the following:
  • Patient is tolerating therapy, AND
  • Patient has experienced a benefit from therapy (e.g., disease amelioration compared to untreated patients); AND
• Prescribed by or in consultation with a neurologist with expertise in the treatment of DMD; AND
• Dose does not exceed 80 mg/kg of body weight infused once weekly; AND
• Submission of medical records (e.g., chart notes, laboratory values) documenting the patient is maintaining ambulatory status, as evaluated via the 6-minute walk test (6MWT) or North Star ambulatory assessment (NSAA).

This Medication Is Not Approvable for the Following Condition(s):

The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics Committee.

Dosing:

• 80 milligrams per kilogram (mg/kg) of body weight once weekly.
  • Viltepso is available as 250 mg/5 mL (50 mg/mL) in a single-dose vial.

Approval:

• Initial: up to 6 months.
• Reauthorization: up to 12 months.

Additional Information:

• Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Review History:

• 04/20/2021- New policy.

References:

• North Star Ambulatory Assessment (2018). https://www.touchneurology.com/wp-content/uploads/sites/3/2018/03/www.musculardystrophyuk.org_assets_0000_6388_NorthStar.pdf.   Accessed April 6, 2021.
• ATS Committee on Proficiency Standards for Clinical Pulmonary Function Laboratories. ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med. 2002 Jul 1;166(1):111-7. doi: 10.1164/ajrccm.166.1.at1102. Erratum in: Am J Respir Crit Care Med. 2016 May 15;193(10):1185. PMID: 12091180. https://www.atsjournals.org/doi/pdf/10.1164/ajrccm.166.1.at1102
• Erratum: ATS Statement: Guidelines for the Six-Minute Walk Test. Am J Respir Crit Care Med. 2016 May 15;193(10):1185. doi: 10.1164/rccm.19310erratum. Erratum for: Am J Respir Crit Care Med. 2002 Jul 1;166(1):111-7. PMID: 27174486. https://www.atsjournals.org/doi/10.1164/rccm.19310erratum
• Holland AE, Spruit MA, Troosters T, Puhan MA, Pepin V, Saey D, McCormack MC, Carlin BW, Sciurba FC, Pitta F, Wanger J. An official European Respiratory Society/American Thoracic Society technical standard: field walking tests in chronic respiratory disease. European Respiratory Journal. 2014 Dec 1;44(6):1428-46. https://erj.ersjournals.com/content/erj/44/6/1428.full.pdf
• Viltepso [package insert], Paramus, NJ: NS Pharma, Inc.; August 2020