WHA
SKYCLARYS (omaveloxolone)

Self-Administration – oral capsule

 

Diagnosis considered for coverage:

 

  • Friedreich's ataxia: Indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. 

 

Coverage Criteria:

 

For diagnosis of Friedreich's ataxia:

  • Diagnosis of Friedreich's ataxia confirmed via genetic testing demonstrating mutation in the FXN gene; AND
  • Patient is 16 years of age or older; AND
  • Patient has a Modified Friedreich's Ataxia Rating Scale (mFARS) score of greater than or equal to 20 and less than or equal to 80; AND
  • Patient has a B-type natriuretic peptide (BNP) value less than or equal to 200 pg/mL; AND
  • Prescribed by or in consultation with one of the following:
    • Neurologist
    • Neurogeneticist
    • Physiatrist (Physical Medicine and Rehabilitation Specialist)

 

Reauthorization Criteria:

 

For diagnosis of Friedreich's ataxia:

  • Documentation of positive clinical response to therapy; AND
  • Patient has a Modified Friedreich's Ataxia Rating Scale (mFARS) score of less than or equal to 80.

 

Dosing:

 

  • 150 mg (3 capsules) taken orally once daily.

 

Coverage Duration:

 

  • 1 year

 

Authorization is not covered for the following:
  • 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 (P&T) Committee.

 

Additional Information:
  • Skyclarys (omaveloxolone) is a nuclear factor erythroid 2-related factor 2 (Nrf2) activator. It was granted fast track and priority review with orphan drug and rare pediatric disease designations as the first FDA-approved treatment for Friedreich’s ataxia (FA).
  • The Modified Friedreich’s Ataxia Rating Scale (mFARS) is a neurological exam-based rating scale that is used as an outcome in trials and is a modified version of the FARS developed in 2003 a quantifiable measure of functional abilities in relation to neurological symptoms of FA. The mFARS has 4 subsections (bulbar, upper limb coordination, lower limb coordination, and upright stability). Scores range from 0 to 93, with lower scores indicating better neurological function.
  • Patients enrolled in the trial were those with an mFARS score between 20 and 80. There is no evidence of benefit for patients with severe neurologic dysfunction with an mFARS score of greater than 80.
  • Instructions for administering the Friedreich’s Ataxia Rating Scale (FARS) and Modified FARS (mFARS): https://www.curefa.org/pdf/Instructions-for-administering-the-mFARS.pdf
  • A B-type natriuretic peptide (BNP) test is a blood test that measures the levels of a protein called BNP that is made by the heart and blood vessels. When the heart has to work harder to pump blood, it makes more BNP. Higher levels of BNP can be a sign of heart failure. While some researchers claim that values above 100 pg/ml indicate heart failure, others suggest a value above 200 pg/ml. A related test, called the N-terminal pro-BNP test, is done in the same way. It provides similar information, but the normal range is different.

 

Policy Updates:
  • 09/01/2023 – New program for Skyclarys approved by P&T. (P&T 08/15/2023)

 

References:
  1. Corben LA, Collins V, Milne S, et al. Clinical management guidelines for Friedreich ataxia: best practice in rare diseases. Orphanet J Rare Dis. 2022 Nov 12;17(1):415.
  2. Lynch DR, Chin MP, Boesch S, et al. Efficacy of omaveloxolone in Friedreich's ataxia: delayed-start analysis of the moxie extension. Mov Disord. 2023;38(2):313-320.
  3. Lynch DR, Chin MP, Delatycki MB, et al. Safety and efficacy of omaveloxolone in Friedreich Ataxia (MOXIe Study). Ann Neurol. 2021;89(2):212-225.
  4. Lynch DR, Farmer J, Hauser L, et al. Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia. Ann Clin Transl Neurol. 2019;6(1):15-26.
  5. Rummey C, Zesiewicz TA, Perez-Lloret S, et al. Test-retest reliability of the Friedreich's ataxia rating scale. Ann Clin Transl Neurol. 2020;7(9):1708-1712.
  6. Skyclarys Package Insert. Reata Pharmaceuticals, Inc. Plano, TX. March 2023.
  7. U.S. Food and Drug Administration (FDA). Summary review. February 28, 2023. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/216718Orig1s000SumR.pdf. Accessed March 28, 2023.
  8. U.S. Food and Drug Administration. (2023, February 28). FDA approves first treatment for Friedreich’s ataxia. Retrieved from https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-first-treatment-friedreichs-ataxia. Accessed June 13, 2023.

Last review date: September 1, 2023