CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

deflazacort (Emflaza) for Duchenne muscular dystrophy

Covers Emflaza (brand deflazacort) and generic deflazacort for treatment of Duchenne muscular dystrophy (DMD) when specified clinical and documentation criteria are met; other indications are considered experimental/investigational and not medically necessary.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

Policydeflazacort (Emflaza) for Duchenne muscular dystrophy

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

Key ActionSubmit laboratory confirmation of DMD diagnosis (genetic testing or muscle biopsy) and chart documentation of prior prednisone/prednisolone adverse effects to initiate prior authorization review.

POLICY UPDATE CHANGES

No material changes to clinical coverage criteria.

2Brand vs Generic age indications

6mInitial authorization length

12mContinuation authorization length

1Covered condition (DMD)

Coverage Summary

Covers Emflaza (brand deflazacort) and generic deflazacort for treatment of Duchenne muscular dystrophy (DMD) when specified clinical and documentation criteria are met; all other indications are considered experimental/investigational and not medically necessary.

Policy thresholds

Age threshold for Emflaza>= 2 years

Age threshold for generic deflazacort>= 5 years

Initial authorization duration6 months

Continuation authorization duration12 months

BMI/weight status for children (percentile)Overweight: 85th to <95th percentile; Obese: >=95th percentile

BMI for adults (kg/m2)Overweight: 25.0-29.9; Obese: >=30.0

Prescriber Specialty & Site Requirements

Documentation Required

Prescriber specialty requirement

Ensure the prescription is written by a physician who specializes in the treatment of Duchenne muscular dystrophy (DMD) or is written in consultation with such a specialist.

Provider Actions & Prior Authorization

Prior Authorization

Prior authorization submission required

Submit prior authorization request with laboratory confirmation of DMD diagnosis (genetic testing or muscle biopsy) and chart documentation of prior prednisone or prednisolone adverse effects (weight gain/obesity or persistent psychiatric/behavioral issues) to initiate PA review.

Documentation Required

Evidence of prior corticosteroid intolerance

Document a trial of prednisone or prednisolone that resulted in unmanageable and clinically significant weight gain/obesity or psychiatric/behavioral issues (examples: abnormal behavior, aggression, irritability).

Prior Authorization

Authorization durations

Initial authorizations are granted for 6 months. Continuation authorizations may be granted for 12 months contingent on meeting continuation criteria, including that the member continues to meet initial criteria and is receiving documented clinical benefit from therapy (e.g., improvement or stabilization of muscle strength or pulmonary function).

Clinical Evidence & References

Primary product labeling and guideline references: Emflaza package insert (June 2021); Deflazacort package inserts (June 2024 and April 2024); Lancet Neurol (2010, 2018) diagnosis and management guidance; American Academy of Neurology practice guideline update (2016); additional DMD management and clinical trial references as cited.

Definitions

Documented DMD diagnosisGenetic testing demonstrating a mutation in the DMD gene or muscle biopsy demonstrating absent dystrophin

Clinical benefitImprovement or stabilization of muscle strength or pulmonary function while receiving therapy

Background

Emflaza and generic deflazacort are corticosteroids indicated for the treatment of Duchenne muscular dystrophy. Emflaza (brand) is FDA‑approved for patients 2 years of age and older; generic deflazacort products are indicated for patients 5 years of age and older. The policy requires laboratory confirmation of DMD diagnosis (genetic testing or muscle biopsy) and chart documentation of intolerance to prednisone or prednisolone (unmanageable weight gain/obesity or persistent psychiatric/behavioral issues) prior to coverage authorization.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

Policydeflazacort (Emflaza) for Duchenne muscular dystrophy

Policy CodePolicy N/A

Change TypeNo material change

Effective Date

Next Review Date

On This Page

OpenPayer

deflazacort (Emflaza) for Duchenne muscular dystrophy

Coverage Summary

FDA‑Approved Indications / Covered Indication

Initial Therapy Criteria (Initial authorization - 6 months)

Continuation Therapy Criteria

Prescriber Specialty & Site Requirements

Provider Actions & Prior Authorization

Unproven / Exclusions

Applicable Codes

Clinical Evidence & References

Background