CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Emflaza (deflazacort) for Duchenne muscular dystrophy — Coverage Criteria

Defines coverage and prior authorization requirements for Emflaza (deflazacort) for treatment of Duchenne muscular dystrophy (DMD) for plan members, including initial and continuation authorization criteria and required documentation.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEmflaza (deflazacort) for Duchenne muscular dystrophy — Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material change

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization with documentation of DMD confirmation and intolerance to prednisone/prednisolone.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

≥2 yrsage indication

6 moinitial auth

12 mocontinuation auth

genetic/biopsydiagnostic confirmation

intolerance requiredprior steroid trial

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inv-01: Initial therapy for DMD

Covered when ALL of the following are met:

Initial authorization criteria: (1) Diagnosis of Duchenne muscular dystrophy (DMD) confirmed by genetic testing demonstrating a mutation in the DMD gene OR muscle biopsy demonstrating absent dystrophin; (2) Patient is 2 years of age or older; (3) Patient has tried prednisone or prednisolone and experienced unmanageable and clinically significant weight gain/obesity OR persistent psychiatric/behavioral issues (for psychiatric/behavioral issues these persisted beyond the first 6 weeks of treatment; for weight gain/obesity body mass index is in the overweight or obese category while receiving prednisone or prednisolone per Appendix).age >= 2 years; weight/BMI thresholds per Appendix

Authorization of up to 6 months may be granted when criteria are met.

inv-02: Continuation therapy

Covered when ALL of the following are met:

Continuation authorization criteria: (1) Member meets all initial authorization criteria; (2) Member is receiving clinical benefit from Emflaza, such as improvement or stabilization of muscle strength or pulmonary function.

Authorization of up to 12 months may be granted.

All indications for Emflaza (deflazacort) other than treatment of Duchenne muscular dystrophy (DMD) are considered experimental/investigational and not medically necessary. This policy does not authorize coverage for Emflaza when used for diagnoses outside the FDA‑approved DMD indication.

Use of Emflaza for non‑DMD indications is not medically necessary. Providers should not expect coverage for Emflaza when prescribed for conditions other than DMD, as such uses are considered experimental/investigational.

Provider Actions and Prior Authorization Requirements

Prior Authorization

Prior Authorization Required

Prior authorization required. Initial authorization may be granted for 6 months when diagnostic confirmation of Duchenne muscular dystrophy (DMD) is provided (genetic test or muscle biopsy).

Initial auth: up to 6 months
Diagnosis: genetic testing demonstrating a mutation in the DMD gene OR muscle biopsy demonstrating absent dystrophin
Age: member is 2 years of age or older

Prior Authorization

Continuation Authorization

Continuation authorization may be granted for 12 months when the member meets initial authorization criteria and is receiving clinical benefit from Emflaza (e.g., improvement or stabilization of muscle strength or pulmonary function).

Continuation auth: up to 12 months
Member must meet all initial authorization criteria
Clinical benefit must be documented (improvement or stabilization of muscle strength or pulmonary function)

Prior Authorization

Required Prior Corticosteroid Trial

Member must have tried prednisone or prednisolone and experienced unmanageable/clinically significant weight gain/obesity or persistent psychiatric/behavioral adverse effects.

Required prior trial: prednisone or prednisolone
For weight gain/obesity: BMI in the overweight or obese category while receiving prednisone/prednisolone (see appendix for weight status categories)
For psychiatric/behavioral issues: symptoms persisted beyond the first 6 weeks of prednisone/prednisolone treatment

Documentation Required

Required Documentation for Prior Authorization

Submit chart documentation to support the prior authorization request.

Documentation must include confirmation of DMD by genetic testing or muscle biopsy
Provide chart documentation of weight gain/obesity or persistent psychiatric/behavioral issues while on prior prednisone or prednisolone therapy

Denial Risk

Non‑approved Indications

Use of Emflaza for indications other than FDA‑approved treatment of DMD is considered experimental/investigational and not medically necessary; such requests may be denied.

FDA‑approved indication: treatment of Duchenne muscular dystrophy in patients 2 years of age and older
All other indications are considered experimental/investigational and not medically necessary

Background

Duchenne muscular dystrophy (DMD) is the FDA‑approved indication for Emflaza; the product is indicated for treatment of DMD in patients aged 2 years and older. DMD is a progressive X‑linked neuromuscular disorder caused by mutations in the DMD gene leading to absent or deficient dystrophin, resulting in progressive muscle weakness and loss of function. Standard corticosteroid therapy (prednisone or prednisolone) is commonly used, but some patients experience intolerable adverse effects (for example, clinically significant weight gain/obesity or persistent psychiatric/behavioral issues) that may prompt consideration of deflazacort (Emflaza) under the coverage criteria outlined in this policy.

Definitions and Appendix

Weight status categories — Definitions for documenting weight gain

Pediatric categories (use for children)Underweight: <5th percentile; Normal/Healthy weight: 5th–<85th percentile; Overweight: 85th–<95th percentile; Obese: ≥95th percentile.

Adult BMI cutoffs (use for adults)Underweight: <18.5; Normal/Healthy weight: 18.5–24.9; Overweight: 25.0–29.9; Obese: ≥30.0.

Use in prior authorizationA patient is considered to have unmanageable/clinically significant weight gain if BMI or pediatric percentile places them in the overweight or obese category while receiving prednisone or prednisolone (refer to Appendix).

Reference for documentationProviders must document BMI or pediatric percentile on prednisone/prednisolone to support intolerance when requesting Emflaza; see Appendix categories for classification.

OpenPayer

Emflaza (deflazacort) for Duchenne muscular dystrophy — Coverage Criteria

Coverage Criteria for Emflaza (deflazacort)

Coding and Weight/BMI Appendix

Provider Actions and Prior Authorization Requirements

Background

Definitions and Appendix