CurrentNeighborhood Health Plan of Rhode IslandPolicy 1636-A

Deflazacort (Emflaza, generics) for Duchenne muscular dystrophy — Prior authorization criteria

Covers prior authorization criteria and continuation requirements for deflazacort products for treatment of Duchenne muscular dystrophy (DMD) when approval criteria are met; applies to prescribers and members covered by Neighborhood Health Plan of Rhode Island.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyDeflazacort (Emflaza, generics) for Duchenne muscular dystrophy — Prior authorization criteria

Policy CodePolicy 1636-A

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionSubmit prior authorization with laboratory confirmation of DMD and documentation of intolerance to prednisone/prednisolone to request deflazacort.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

2 yrsmin age Emflaza

5 yrsmin age other deflazacort

6 moinitial auth

12 mocontinuation auth

BMI-basedweight criteria

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Coverage Criteria for Deflazacort (DMD)

Initial Therapy

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

Initial authorization criteria: Diagnosis of Duchenne muscular dystrophy (DMD) confirmed by genetic testing demonstrating a mutation in the DMD gene OR muscle biopsy demonstrating absent dystrophin.N/A

Cite laboratory confirmation (genetic test or muscle biopsy).

Age requirement: Member is age‑eligible for the requested deflazacort product: Emflaza (deflazacort brand) — >= 2 years; other deflazacort products (generic, Kymbee, Jaythari, Pyquvi) — >= 5 years.age per product labeling

Use product-specific minimum ages.

Prior corticosteroid intolerance/step therapy: Member has tried prednisone or prednisolone and experienced unmanageable and clinically significant weight gain/obesity OR psychiatric/behavioral issues (for example, abnormal behavior, aggression, irritability) while receiving prednisone/prednisolone.For weight-related intolerance, BMI must be in the overweight or obese category while on prednisone/prednisolone (see Appendix BMI percentiles for children and BMI cutoffs for adults).

Document chart evidence of adverse effects during prednisone/prednisolone therapy.

Continuation Therapy

Authorization of 12 months may be granted for continuation when ALL of the following are met:

Continuation criteria: Member meets all initial coverage requirements for DMD treatment with deflazacort and the prescriber documents that the member is receiving clinical benefit from the requested medication (examples: improvement or stabilization of muscle strength or pulmonary function).Documented clinical benefit required for continuation.

Continuation authorizations may be granted for 12 months when benefit is shown.

Not Medically Necessary / Experimental Uses

Indications not covered: Any indication other than Duchenne muscular dystrophy (including non–FDA‑approved or off‑label uses) is considered experimental/investigational and not medically necessary.N/A

Requests for deflazacort for non‑DMD indications will be denied.

All uses of deflazacort products outside of the specified Duchenne muscular dystrophy (DMD) indications are considered experimental/investigational and not medically necessary. Authorization and coverage apply only when the indication is DMD as described in this policy.

Use of deflazacort (including branded and generic formulations) for any indication other than Duchenne muscular dystrophy is not medically necessary and is not covered under this policy.

Drug Products and Coding References

Covered drug products referencedNDC | brandedCovered

Emflaza	deflazacort (brand)
deflazacort (generic)	deflazacort (generic)
Kymbee	deflazacort product
Jaythari	deflazacort product
Pyquvi	deflazacort product

Age and BMI thresholds — Emflaza and other deflazacort products

Minimum age — EmflazaEmflaza is indicated for patients 2 years of age and older.

Minimum age — other deflazacort productsDeflazacort (generic), Kymbee, Jaythari, and Pyquvi are indicated for patients 5 years of age and older.

BMI definitions referenceBMI categories and percentiles used to assess weight-related intolerance are defined in the Appendix (children and adults).

Provider Requirements and Authorization Actions

Prior Authorization

Initial Authorization — PA required

Initial prior authorization is required. Authorization of up to 6 months may be granted for treatment of Duchenne muscular dystrophy (DMD) when the diagnosis is confirmed, the member is age‑eligible, and step therapy and documentation requirements are met.

Authorization duration: up to 6 months for initial therapy.
Step therapy: member must have tried prednisone or prednisolone and experienced unmanageable and clinically significant weight gain/obesity or persistent psychiatric/behavioral issues (e.g., abnormal behavior, aggression, irritability).
Age eligibility: member is 2 years of age or older for Emflaza; deflazacort generics/brands indicated for ages 5+ (per FDA labeling).
Required diagnostic confirmation: laboratory confirmation of DMD by genetic testing or muscle biopsy.
Required documentation: chart documentation of weight gain/obesity (BMI in overweight/obese category while on prednisone/prednisolone) or persistent psychiatric/behavioral issues with prior prednisone/prednisolone treatment.
Prescriber specialty: must be prescribed by or in consultation with a physician who specializes in the treatment of DMD.
Missing documentation risk: lack of laboratory confirmation of DMD (genetic testing or muscle biopsy) may trigger denial.

Background

Deflazacort is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD). Emflaza is FDA‑approved for patients 2 years of age and older; the generic deflazacort products (deflazacort, Kymbee, Jaythari, Pyquvi) are FDA‑approved for patients 5 years of age and older. Coverage under this policy is limited to these FDA‑approved DMD indications.

Definitions and Clinical Terms

Confirmed Duchenne muscular dystrophy (DMD)

DefinitionDiagnosis confirmed by genetic testing demonstrating a mutation in the DMD gene OR muscle biopsy demonstrating absent dystrophin.

Source contextRequired as part of initial authorization criteria for treatment of Duchenne muscular dystrophy.

Documentation noteLaboratory confirmation (genetic testing) or muscle biopsy results must be submitted for prior authorization review.

Weight status categories (BMI) used to assess prednisone/prednisolone intolerance

Children (2–19 years) — OverweightBMI percentile 85th to less than 95th percentile = Overweight.

Children (2–19 years) — ObeseBMI percentile equal to or greater than the 95th percentile = Obese.