CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Emflaza 1636 A Sgm P2022

Covers Emflaza for treatment of Duchenne muscular dystrophy (DMD) in patients aged 2 years and older when prior authorization criteria are met; all other indications are considered experimental/investigational and not medically necessary. Defines documentation required for prior authorization and criteria for initial and continuation approvals.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEmflaza 1636 A Sgm P2022

Policy CodePolicy N/A

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionSubmit prior authorization with laboratory confirmation of DMD (genetic testing or muscle biopsy) and chart documentation of prior prednisone/prednisolone–related unmanageable weight gain/obesity or persistent psychiatric/behavioral issues.

SourceLink

POLICY UPDATE CHANGES

No material changes — policy has no listed changes.

1Covered Indication (DMD)

>=2 yrsMinimum age

6Initial auth (months)

12Continuation auth (months)

Not covered

Coverage Summary

Covers Emflaza (deflazacort) for the FDA-approved treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older when prior authorization criteria are met. All other indications are considered experimental/investigational and not medically necessary.

Prior authorization requires documentation of diagnostic confirmation (genetic testing demonstrating a DMD gene mutation or muscle biopsy demonstrating absent dystrophin) and chart documentation of prior use of prednisone or prednisolone with resulting unmanageable and clinically significant weight gain/obesity or persistent psychiatric/behavioral issues to justify switching to Emflaza. Initial approvals may be granted for 6 months when criteria are met; continuation approvals may be granted for 12 months with documentation of ongoing clinical benefit (for example improvement or stabilization of muscle strength or pulmonary function).

Weight status categories for children and adults are provided as reference for assessing steroid-related weight gain: pediatric BMI percentiles defining Overweight: 85th to <95th percentile and Obese: ≥95th percentile; adult BMI categories defining Overweight: 25.0–29.9 and Obese: ≥30.0.

Key Thresholds

Minimum age for therapy>= 2 years

Initial authorization duration6 months

Continuation authorization duration12 months

Required prior steroid trialTrial of prednisone or prednisolone with documented unmanageable weight gain/obesity or persistent psychiatric/behavioral issues

Psychiatric/behavioral persistence thresholdPersisted beyond the first 6 weeks of prednisone/prednisolone treatment

Non-covered usesAll indications other than FDA-approved DMD are experimental/not medically necessary

Initial Therapy Criteria (Authorization — 6 months)

Authorization of 6 months may be granted for treatment of DMD when all of the following criteria are met:

ALL of the following

Diagnosis confirmed (ONE of)
- Genetic testing demonstrating a mutation in the DMD gene.
- Muscle biopsy demonstrating absent dystrophin.
The member is 2 years of age or older.
Prior trial of prednisone/prednisolone with ONE of

Continuation Therapy Criteria (Authorization — 12 months)

Unproven / Non‑covered Indications

Provider Actions / Prior Authorization & Documentation

Prior Authorization

Prior authorization required

Submit prior authorization with documentation including laboratory confirmation of DMD by genetic testing or muscle biopsy, and chart documentation of weight gain/obesity or persistent psychiatric/behavioral issues with prior prednisone or prednisolone treatment.

Documentation Required

Required diagnostic confirmation

Provide genetic testing demonstrating a DMD gene mutation or muscle biopsy demonstrating absent dystrophin.

Applicable Codes

No codes specified in policymixed

No codes listed

Clinical Evidence & References

Primary reference: Emflaza [package insert], June 2021.

Supporting guidelines and trials: Lancet Neurol 2010; AAN guideline update 2016; Neurology 2016 trial comparing deflazacort vs prednisone.

Background & Definitions

Emflaza is FDA-approved for treatment of Duchenne muscular dystrophy in patients 2 years of age and older.

The policy requires prior trial with prednisone or prednisolone and documentation of steroid-related adverse effects to justify switching to Emflaza: either clinically significant weight gain/obesity while receiving prednisone/prednisolone (per the provided BMI percentile or BMI categories) or psychiatric/behavioral issues that persisted beyond the first 6 weeks of prednisone/prednisolone treatment. Diagnostic confirmation of DMD by genetic testing or muscle biopsy is required for authorization.

Weight status category tables are included in the policy as reference: pediatric BMI percentiles (Overweight = 85th to <95th percentile; Obese = ≥95th percentile) and adult BMI ranges (Overweight = 25.0–29.9; Obese = ≥30.0).

BMI Categories

Overweight (children)BMI percentile 85th to <95th

Obese (children)BMI percentile >=95th

Overweight (adults)BMI 25.0 - 29.9

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