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Skyclarys (omaveloxolone) Prior Authorization with Quantity Limit Program Summary
Policy Number: PH-1207
This program applies to Blue Partner, Commercial, GenPlus, NetResults A series, SourceRx and Health Insurance Marketplace formularies.
POLICY REVIEW CYCLE
Effective Date |
Date of Origin |
07-01-2024 |
10-01-2023 |
FDA LABELED INDICATIONS AND DOSAGE
Agent(s) |
FDA Indication(s) |
Notes |
Ref# |
Skyclarys® (omaveloxolone) Capsule |
Treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older |
|
1 |
See package insert for FDA prescribing information: https://dailymed.nlm.nih.gov/dailymed/index.cfm
CLINICAL RATIONALE
Friedreich Ataxia |
Friedreich ataxia (FA, FRDA) is a progressive autosomal recessive genetic neurodegenerative disorder affecting approximately 5,000 patients in the United States and 22,000 patients globally.(4,5) FA is caused by a biallelic trinucleotide (GAA) repeat expansion in the first intron of the FXN gene, which impairs transcription and significantly reduces the amount of functional frataxin protein. The pathological consequences of frataxin deficiency include disruption of iron–sulfur cluster biosynthesis, cellular iron dysregulation, mitochondrial dysfunction, and increased sensitivity to oxidative stress leading to the clinical features of FA.(2,3,4,5) Ataxia is the most common clinical feature in FA, reflecting both proprioceptive loss and cerebellar disease. Patients can also develop spasticity, visual and hearing loss, and non‐neurological features such as cardiomyopathy, diabetes, and scoliosis. In most patients, symptoms begin between 5 and 15 years of age, and patients lose the ability to ambulate by their mid‐20s. FA shortens life span, most often through consequences of cardiomyopathy; average age at death is 37 years.(4,5) Genetic testing for the triplet repeat expansions in the first intron of the frataxin (FXN) gene that cause Friedreich ataxia should be performed in all patients with progressive cerebellar ataxia and autosomal recessive inheritance.(2,3,4,5) In individuals who are ambulant, clinical management guidelines recommend regular monitoring of ambulation and contributing physical and non-physical factors for mobility decline (e.g., balance, strength, lower limb spasticity, fear of falling) at least once per year. For non-ambulant individuals, regular monitoring of mobility (including ability to transfer) and contributing factors for mobility decline (e.g., balance, strength, lower limb spasticity, environmental set-up) is recommended at least once per year.(6) Until omaveloxolone was approved by the FDA for treatment of FA, there was no specific disease-modifying therapy available. The management of patients with this disorder requires a multidisciplinary team of special services. An occupational and physical therapy program should be initiated early. Periodic evaluation of cardiac function is required. Similarly, patients should be monitored for the development of dysphagia, scoliosis, vision loss, hearing loss, bladder dysfunction, sleep apnea, and diabetes mellitus.(4,5) |
Efficacy |
Omaveloxolone is an activator of the Nuclear factor-like (Nrf2) pathway, which is involved in the cellular response to oxidative stress.(1) Treatment with omaveloxolone in vitro restores mitochondrial function in fibroblasts from Friedreich ataxia patients and in neurons from multiple mouse models.(4,5) In a larger international randomized trial, 103 patients with Friedreich ataxia (median age, 21 to 22 years; mean disease duration, approximately 4.5 years) were randomly assigned to omaveloxolone 150 mg daily or placebo for 48 weeks. Efficacy data were presented for 82 patients (80%) who received 48 weeks of treatment and had completed primary outcome measurements on the mFARs. Among these patients, mFARS scores improved by 1.55 points in the omaveloxolone group and worsened by 0.85 points in the placebo group (mean difference between groups -2.4 points, 95% CI -4.3 to -0.5). Adverse effects that occurred more commonly with omaveloxolone than placebo included elevated aminotransferase levels (37 versus 2%; no cases of clinical liver injury), headache (37 versus 25%), and nausea (33 versus 14%).(1) Although the trial had limitations and the effect size was relatively modest, Friedreich ataxia is a slowly progressive disease, and small differences in functional progression over one to two years could translate to meaningful differences over the course of the disease.(5) |
REFERENCES
Number |
Reference |
1 |
Skyclarys prescribing information. Reata Pharmaceuticals, Inc. February 2023. |
2 |
Rummey C, Corben LA, Delatycki M, et al. Natural History of Friedreich Ataxia. Neurology. 2022 Oct;99(14):e1499-e1510. |
3 |
Pandolfo M. Friedreich Ataxia. Arch Neurol. 2008 Oct;65(10):1296-1303. |
4 |
Lynch DR, Chin MP, Delatycki MB, et al. Safety and Efficacy of Omaveloxolone in Friedreich Ataxia (MOXIe Study). Ann Neurol. 2021 Feb;89(2):212-225. |
5 |
Opal P, Zoghbi H, et al. Friedreich Ataxia. UpToDate. Last updated November 2023. Literature review current through December 2023. |
6 |
Corben LA, Collins V, Milne S, et al. Clinical Management Guidelines for Friedreich Ataxia: Best Practice in Rare Diseases. Orphanet J Rare Dis. 2022;17:415. |
POLICY AGENT SUMMARY PRIOR AUTHORIZATION
Target Brand Agent(s) |
Target Generic Agent(s) |
Strength |
Targeted MSC |
Available MSC |
Final Age Limit |
Preferred Status |
|
||||||
Skyclarys |
omaveloxolone cap |
50 MG |
M ; N ; O ; Y |
N |
|
|
POLICY AGENT SUMMARY QUANTITY LIMIT
Target Brand Agent Name(s) |
Target Generic Agent Name(s) |
Strength |
QL Amount |
Dose Form |
Day Supply |
Duration |
Addtl QL Info |
Allowed Exceptions |
Targeted NDCs When Exclusions Exist |
|
|||||||||
Skyclarys |
omaveloxolone cap |
50 MG |
90 |
Capsules |
30 |
DAYS |
|
|
|
CLIENT SUMMARY – PRIOR AUTHORIZATION
Target Brand Agent Name(s) |
Target Generic Agent Name(s) |
Strength |
Client Formulary |
Skyclarys |
omaveloxolone cap |
50 MG |
Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx |
CLIENT SUMMARY – QUANTITY LIMITS
Target Brand Agent Name(s) |
Target Generic Agent Name(s) |
Strength |
Client Formulary |
Skyclarys |
omaveloxolone cap |
50 MG |
Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx |
PRIOR AUTHORIZATION CLINICAL CRITERIA FOR APPROVAL
Module |
Clinical Criteria for Approval |
||
|
Initial Evaluation Target Agent(s) will be approved when ALL of the following are met:
Length of Approval: 12 months NOTE: If Quantity Limit applies, please refer to Quantity Limit Criteria.
Renewal Evaluation
Length of Approval: 12 months NOTE: If Quantity Limit applies, please refer to Quantity Limit Criteria. |
QUANTITY LIMIT CLINICAL CRITERIA FOR APPROVAL
Module |
Clinical Criteria for Approval |
|
Quantity limit for the Target Agent(s) will be approved when ONE of the following is met:
Length of Approval: up to 12 months |
This pharmacy policy is not an authorization, certification, explanation of benefits or a contract. Eligibility and benefits are determined on a case-by-case basis according to the terms of the member’s plan in effect as of the date services are rendered. All pharmacy policies are based on (i) information in FDA approved package inserts (and black box warning, alerts, or other information disseminated by the FDA as applicable); (ii) research of current medical and pharmacy literature; and/or (iii) review of common medical practices in the treatment and diagnosis of disease as of the date hereof. Physicians and other providers are solely responsible for all aspects of medical care and treatment, including the type, quality, and levels of care and treatment.
The purpose of Blue Cross and Blue Shield of Alabama’s pharmacy policies are to provide a guide to coverage. Pharmacy policies are not intended to dictate to physicians how to practice medicine. Physicians should exercise their medical judgment in providing the care they feel is most appropriate for their patients.
Neither this policy, nor the successful adjudication of a pharmacy claim, is guarantee of payment.
Commercial _ PS _ Skyclarys_omaveloxolone__PAQL _ProgSum_ 07-01-2024