ModifiedHealth NetPolicy CP.PHAR.331

Deflazacort (Emflaza) coverage for Duchenne muscular dystrophy

Defines medical necessity criteria, prior authorization and continuation requirements for deflazacort (Emflaza) for members with Duchenne muscular dystrophy (DMD) across Health Net/Centene lines of business.

Policy Summary

PayerHealth Net

PolicyDeflazacort (Emflaza) coverage for Duchenne muscular dystrophy

Policy CodePolicy CP.PHAR.331

Change TypeOperational additions and clarifications

Effective DateApr 1, 2017

Next Review DateN/A

Key ActionSubmit prior authorization with documentation including member's current weight in kg and evidence of prior prednisone trial or contraindication.

SourceLink

POLICY UPDATE CHANGES

Added requirement for documentation of member's current weight in kg to initial and continued approval criteria.

Revised policy/criteria to include use of generic deflazacort for Emflaza requests unless contraindicated or adverse effects occur.

Commercial continued approval authorization changed to 12 months or duration of request, whichever is less.

0.9 mg/kg/daymaximum daily dose for DMD

≥2 yrsFDA-approved minimum age

6 moInitial approval duration

12 mo

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Coverage and Medical Necessity Criteria

Initial Therapy

Initial Approval — Covered when ALL of the following are met:

Initial Approval Criteria: 1) Diagnosis of Duchenne muscular dystrophy confirmed by genetic testing showing dystrophin deletion or duplication OR, if genetic studies are negative, positive muscle biopsy demonstrating absence of dystrophin protein; 2) Prescribed by or in consultation with a neurologist; 3) Age ≥ 2 years; 4) Failure of a ≥ 6 month trial of prednisone unless prednisone is contraindicated or causes clinically significant adverse effects; 5) For Emflaza requests, member must use generic deflazacort unless contraindicated or clinically significant adverse effects are experienced; 6) Documentation of member's current weight in kg; 7) Dose does not exceed 0.9 mg/kg per day.

Approval duration: 6 months

Continuation Therapy

Continued Therapy — Covered when ALL of the following are met:

Continuation Criteria: 1) Member is currently receiving medication via Centene benefit or has previously met initial approval criteria OR member is enrolled in a state/product with continuity of care regulations; 2) Member is responding positively to therapy; 3) For Emflaza requests, member must use generic deflazacort unless contraindicated or clinically significant adverse effects are experienced; 4) Documentation of member's current weight in kg; 5) If request is for dose increase, new dose does not exceed 0.9 mg/kg per day.

Authorization duration: Medicaid/HIM 12 months; Commercial 12 months or duration of request, whichever is less

Requests for uses that are not FDA‑approved and are not specifically addressed in this policy are generally not authorized unless the request includes sufficient documentation of safety and efficacy consistent with the applicable off‑label use policies (for example, CP.CPA.09 for Commercial, HIM.PA.154 for the Health Insurance Marketplace, or CP.PMN.53 for Medicaid).

Deflazacort (Emflaza) is contraindicated in individuals with known hypersensitivity to deflazacort or to any of the inactive ingredients in Emflaza.

Coverage determinations for deflazacort (Emflaza) are made in accordance with the Health Plan's clinical policy as a guide to medical necessity but remain subject to the specific terms, conditions, exclusions, and limitations of the member's coverage documents (for example, evidence of coverage, certificate of coverage, policy or contract of insurance) and to applicable state and federal requirements and Health Plan administrative policies.

This policy does not guarantee payment and does not replace any contractual provisions; if a conflict exists between this clinical policy and legal or regulatory requirements, the requirements of law and regulation govern.

Requests for deflazacort for non‑FDA indications that are not addressed in this policy will be considered not authorized (not medically necessary) in the absence of adequate supporting evidence per the applicable off‑label use policies.

Product and Procedure Codes

NDC / Product codesNDC

NDC not listed

No specific NDCs provided in this document

Procedure/Billing codesCPT

HCPCS/CPT not listed

No procedure or HCPCS/CPT codes provided in this document

inv-09: Maximum daily dose

Maximum daily dose0.9 mg/kg/day (dose must not exceed 0.9 mg/kg per day)

Dosing frequency noteDosing regimen for DMD listed as 0.9 mg/kg once daily (QD)

Documentation requiredProvider must document member's current weight in kg to calculate daily dose

Approval implicationRequests for dose increases must remain ≤ 0.9 mg/kg/day for continued approval

What Providers Must Do

Prior Authorization

Prior authorization required for deflazacort/Emflaza

Prior authorization is required for deflazacort/Emflaza. Initial approvals are granted only when the member meets the specified diagnostic and clinical criteria (see Initial Approval) and are limited to a 6-month authorization period.

Initial approval duration: 6 months
Approval requires meeting all Initial Approval criteria (diagnostic confirmation, prescriber/consult with neurologist, age ≥2 years, prior prednisone trial unless contraindicated, use of generic deflazacort for Emflaza requests unless contraindicated, documentation of current weight in kg, dose ≤ 0.9 mg/kg/day).

Note

Prior authorization governed by plan documents

This clinical policy is a medical necessity guide to assist coverage decisions, but prior authorization requirements are governed by the Health Plan's administrative policies and the member's coverage documents.

Coverage and any prior authorization requirements are subject to the plan's evidence of coverage, certificates, contracts, and applicable administrative policies and procedures.

Clinical Background

Duchenne muscular dystrophy (DMD) is an X‑linked genetic disorder caused by mutations that result in absence or deficiency of the structural muscle protein dystrophin, leading to progressive skeletal muscle weakness, loss of ambulation, and premature cardiopulmonary decline. Corticosteroid therapy, including deflazacort, is used to slow loss of muscle strength and function in patients with DMD and is FDA‑approved for this indication in patients aged ≥ 2 years. Clinical use balances potential benefits on strength and function against corticosteroid adverse effects and informs the policy criteria for appropriate prescribing and monitoring.

Diagnostic Confirmation and Definitions

inv-19: DMD diagnostic confirmation

Genetic testing confirmationConfirmed DMD by genetic testing showing dystrophin deletion or duplication mutation

Muscle biopsy confirmationIf genetic studies are negative, diagnosis may be confirmed by positive muscle biopsy showing absence of dystrophin protein

Prescriber requirementPrescription by or in consultation with a neurologist is required for initial approval

Age requirementDiagnosis and treatment applicable for patients age ≥ 2 years per initial criteria