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Emflaza (deflazacort) Prior Authorization with Quantity Limit Program Summary
Policy Number: PH-1038
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 |
1/1/2024 |
|
FDA APPROVED INDICATIONS AND DOSAGE
Agent(s) |
FDA Indication(s) |
Notes |
Ref# |
Emflaza® |
Treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older |
|
1 |
See package insert for FDA prescribing information: https://dailymed.nlm.nih.gov/dailymed/index.cfm
CLINICAL RATIONALE
Duchenne Muscular Dystrophy |
Duchenne muscular dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin that helps keep muscle cells intact. DMD is the most common childhood form of muscular dystrophy as well as the most common of the muscular dystrophies overall with more than 30 known forms. DMD is an X-linked recessive inherited genetic disorder primarily affecting males, although females who carry the defective gene may show some symptoms. Prevalence is 15.9 per 100,000 live male births in the US and 19.5 per 100,000 live male births in the UK. Dystrophin is the protein associated with this affected gene and provides structural stability to skeletal muscles. Mutations in this gene, and subsequent lack of dystrophin in muscle fiber, result in a rapidly progressing disease involving muscle degeneration and weakness. Symptom onset is in early childhood and many children lose the ability to walk by early adolescence. Beyond muscle weakness, other symptoms include enlargement of the calf muscles, lumbar lordosis, and later on cardiomyopathyand poor respiratory function. Until relatively recently, boys with DMD usually did not survive much beyond their teen years. Thanks to advances in cardiac and respiratory care, life expectancy is increasing and many young adults with DMD are surviving into their early 30s. Currently, there is no cure for DMD, and therapies are supportive in nature. Physical therapy, occupational therapy, respiratory care, speech therapy, braces/wheelchairs/contractures and glucocorticoid therapy are among the most common therapies. (2-4) |
Efficacy |
The effectiveness of Emflaza for the treatment of DMD was established in one multicenter, randomized, double-blind, placebo-controlled, 52-week study. 196 male patients between the ages of 5 and 15 years old with documented mutation of the dystrophin gene, onset of weakness before 5 years of age, and serum creatinine kinase activity at least 10 times the upper limit of normal at some stage in their illness were enrolled. Patients were randomized to receive Emflaza (0.9 or 1.2 mg/kg/day), an active comparator, or placebo. After 12 weeks, placebo patients were re-randomized to receive either Emflaza or the active comparator. All patients continued treatment for an additional 40 weeks. Efficacy was evaluated by assessing the change between Baseline and Week 12 in average strength of 18 muscle groups. The change in average muscle strength score between Baseline and Week 12 was significantly greater for the deflazacort 0.9 mg/kg/day dose group than for the placebo group. (p-value 0.017). Although not a pre-specified statistical analysis, compared with placebo, the deflazacort 0.9 mg/kg/day dose group demonstrated at Week 52 the persistence of the treatment effect observed at Week 12.(1) |
Safety |
Emflaza is contraindicated in patients with known hypersensitivity to deflazacort or to any of the inactive ingredients. Instances of hypersensitivity, including anaphylaxis, have occurred in patients receiving corticosteroid therapy.(1) |
REFERENCES
Number |
Reference |
1 |
Emflaza prescribing information. Marathon Pharmaceuticals. June 2021. |
2 |
Duchenne muscular dystrophy (DMD). Muscular Dystrophy Association. (2021, April 29). https://www.mda.org/disease/duchenne-muscular-dystrophy |
3 |
Biggar, W. D., Skalsky, A., & McDonald, C. M. (2022). Comparing deflazacort and prednisone in Duchenne Muscular Dystrophy. Journal of Neuromuscular Diseases, 9(4), 463–476. https://doi.org/10.3233/jnd-210776 |
4 |
U.S. Department of Health and Human Services. Muscular dystrophy. National Institute of Neurological Disorders and Stroke. https://www.ninds.nih.gov/health-information/disorders/muscular-dystrophy |
5 |
Gloss, D., Moxley, R. T., Ashwal, S., & Oskoui, M. (2016). Practice guideline update summary: Corticosteroid treatment of Duchenne muscular dystrophy. Neurology, 86(5), 465–472. https://doi.org/10.1212/wnl.0000000000002337 |
6 |
Reference no longer used |
POLICY AGENT SUMMARY PRIOR AUTHORIZATION
Target Brand Agent(s) |
Target Generic Agent(s) |
Strength |
Targeted MSC |
Available MSC |
Final Age Limit |
Preferred Status |
|
||||||
Emflaza |
deflazacort susp |
22.75 MG/ML |
M ; N ; O ; Y |
N |
|
|
Emflaza |
deflazacort tab |
18 MG ; 30 MG ; 36 MG ; 6 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 |
|
|||||||||
Emflaza |
Deflazacort Tab 18 MG |
18 MG |
30 |
Tablets |
30 |
DAYS |
|
|
|
Emflaza |
Deflazacort Tab 6 MG |
6 MG |
60 |
Tablets |
30 |
DAYS |
|
|
|
CLIENT SUMMARY – PRIOR AUTHORIZATION
Target Brand Agent Name(s) |
Target Generic Agent Name(s) |
Strength |
Client Formulary |
Emflaza |
deflazacort susp |
22.75 MG/ML |
Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx |
Emflaza |
deflazacort tab |
18 MG ; 30 MG ; 36 MG ; 6 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 |
Emflaza |
Deflazacort Tab 18 MG |
18 MG |
Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx |
Emflaza |
Deflazacort Tab 6 MG |
6 MG |
Blue Partner ; Commercial ; GenPlus ; Health Insurance Marketplace ; NetResults A Series ; SourceRx |
PRIOR AUTHORIZATION CLINICAL CRITERIA FOR APPROVAL
Module |
Clinical Criteria for Approval |
PA |
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. *Step therapy requirement may not apply if a prior health plan paid for the medication - documentation of a paid claim may be required.
Renewal 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.
|
QUANTITY LIMIT CLINICAL CRITERIA FOR APPROVAL
Module |
Clinical Criteria for Approval |
QL |
Quantity Limit for the Target Agent(s) will be approved when ONE of the following is met:
Approval Length: 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
BCBSAL _ Commercial _ CSReg _ Emflaza (deflazacort) _PAQL _ProgSum_ 1/1/2024