CurrentCignaPolicy IP0131

Muscular Dystrophy - Deflazacort

Cigna coverage policy governing prior authorization, medical necessity criteria, and product preferences for deflazacort (brand and generic formulations) for treatment of Duchenne muscular dystrophy (DMD) under Cigna-administered health plans.

Policy Summary

PayerCigna

PolicyMuscular Dystrophy - Deflazacort

Policy CodePolicy IP0131

Change TypeCriteria revisions and operational documentation updates

Effective Date

Next Review Date

Key ActionObtain prior authorization and provide documentation including genetic test confirming a pathogenic dystrophin variant and records of prior prednisone/prednisolone use when requesting deflazacort.

SourceLink

POLICY UPDATE CHANGES

Diagnosis confirmation now requires genetic testing with a confirmed pathogenic variant in the dystrophin gene; removal of muscle biopsy requirement.

Initial therapy criteria now require prior trial of prednisone or prednisolone for ≥ 6 months with specified intolerable adverse effects or severe behavioral adverse events prompting dose reduction.

Motor function measures updated: 'time to run or walk 30 feet' replaced with 'time to run or walk 10 meters' and 6-minute walk test added as examples.

Preferred product criteria and branded generics (Jaythari, Pyquvi, Kymbee) were added for Employer Plans with formulary exception language.

Documentation requirement flag ('documentation required') added throughout the criteria.

≥2 yrsminimum age for deflazacort approval

≥5 yrsminimum age for Jaythari/Kymbee/Pyquvi

1 year

Policy Summary

PayerCigna

PolicyMuscular Dystrophy - Deflazacort

Policy CodePolicy IP0131

Change TypeCriteria revisions and operational documentation updates

Effective Date

Next Review Date

SourceLink

On This Page

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 Criteria for Deflazacort (Duchenne Muscular Dystrophy)

FDA-Approved Indication: Duchenne Muscular Dystrophy

Approve for 1 year if the patient meets ONE of the following (A or B):

Overall options

A) Initial Therapy
- i: Patient is ≥ 2 years of age
- ii: Patient's diagnosis of Duchenne Muscular Dystrophy is confirmed by genetic testing with a confirmed pathogenic variant in the dystrophin gene [documentation required]
  Genetic test result must be documented
- iii
  - a
    1: Cushingoid appearance [documentation required]
    2: Central (truncal) obesity [documentation required]
    3: Undesirable weight gain defined as ≥ 10% of body weight increase over a 6-month period [documentation required]≥ 10% body weight increase over 6 months
    4: Diabetes and/or hypertension that is difficult to manage per prescriber [documentation required]
  - b: According to the prescriber, the patient has experienced a severe behavioral adverse event while on prednisone or prednisolone therapy that has or would require a prednisone or prednisolone dose reduction [documentation required]
- iv: Medication is prescribed by or in consultation with a physician who specializes in the treatment of Duchenne muscular dystrophy and/or neuromuscular disorders
- v: Preferred product criteria is met for the product(s) as listed in the preferred product tables (employer-plan specifics) [documentation may be required]
  See Employer Plans section for exceptions
B) Patient is Currently Receiving Deflazacort
- i: Patient is ≥ 2 years of age
- ii: Patient has tried prednisone or prednisolone [documentation required]
- iii: According to the prescriber, the patient has responded to or continues to have improvement or benefit from deflazacort therapy [documentation required]; examples include motor function improvement (e.g., time from supine to standing, time to climb four stairs, time to run or walk 10 meters, 6-minute walk test), improvement in muscle strength, or improved pulmonary function
- iv: Medication is prescribed by or in consultation with a physician who specializes in the treatment of Duchenne muscular dystrophy and/or neuromuscular disorders

Employer-plan preferred product exception: For Employer Plans, Emflaza (brand) may be approved only if patient has tried and cannot take generic deflazacort tablets or other branded generics due to formulation difference in inactive ingredients causing significant allergy or serious adverse reaction [documentation required]; similar requirements apply for oral suspension products.

See Employer Plans preferred product tables for specifics

Deflazacort for any other use is considered not medically necessary.

Use of deflazacort for indications other than Duchenne muscular dystrophy is considered not medically necessary.

Coding and Key Clinical Thresholds

No explicit codes listedmixed

No codes listed

inv-05: Undesirable weight gain — threshold parameter used in criteria (≥ 10% body weight increase over 6 months)

ThresholdUndesirable weight gain defined as ≥ 10% body weight increase over a 6-month period

ContextUsed as one of the significant intolerable adverse effects when considering initiation of deflazacort after prednisone/prednisolone trial

DocumentationDocumentation of weight change over the 6-month period is required

inv-06: Prior steroid trial duration — key parameter: ≥ 6 months of prednisone or prednisolone before initiating deflazacort in most cases

Required durationPrior trial of prednisone or prednisolone for ≥ 6 months

Role in criteria

Provider Actions, Documentation, and Authorization

Prior Authorization

Prior Authorization Required

Prior Authorization is required for benefit coverage of deflazacort. All approvals are provided for one year when criteria are met. Because of the specialized skills required for evaluation and diagnosis of patients treated with deflazacort as well as the monitoring required for adverse events and long-term efficacy, approval requires deflazacort to be prescribed by or in consultation with a physician who specializes in the condition being treated.

Approvals provided for 1 year when criteria are met.
Must be prescribed by or in consultation with a specialist in the condition being treated.

Step Therapy

Provider Actions — Initial Therapy Requirements

Initial therapy generally requires a prior trial of prednisone or prednisolone for ≥ 6 months unless intolerable adverse effects are documented. For initial therapy, patient must be ≥ 2 years and have DMD confirmed by genetic testing; documentation of the trial, intolerable adverse effects, and genetic confirmation must be provided.

Background and Context

Duchenne muscular dystrophy (DMD) is a rare, progressive X-linked disease caused by pathogenic variants in the dystrophin gene that leads to progressive skeletal muscle weakness and decline in respiratory and cardiac function. Deflazacort is a corticosteroid indicated for the treatment of DMD in patients aged ≥ 2 years (with some branded generics labeled for use starting at age ≥ 5 years) and has demonstrated efficacy compared with prednisone and placebo in clinical trials.

Under this policy, deflazacort is a treatment option for DMD when specific documentation and medical necessity criteria are met. Prior authorization is required and approvals are provided for 1 year when criteria are satisfied. Initial therapy generally requires documentation of a prior trial of prednisone or prednisolone for at least ≥ 6 months unless the prescriber documents intolerable adverse effects or severe behavioral adverse events that necessitate switching to deflazacort. Examples of qualifying intolerable adverse effects include Cushingoid appearance, central (truncal) obesity, undesirable weight gain defined as ≥ 10% body weight increase over 6 months, and difficult-to-manage diabetes or hypertension.

Documentation supporting the diagnosis and treatment history is required, including genetic testing confirming a pathogenic dystrophin variant, records of prior steroid use and related adverse effects, and evidence of clinical benefit for members currently receiving deflazacort. For Employer Plans, product-specific preferred product requirements and exception pathways apply.

Definitions

inv-12: Duchenne muscular dystrophy (DMD) — definition and concise disease description

DefinitionDuchenne muscular dystrophy (DMD) is a rare, progressive X-linked disease caused by mutation(s) in the dystrophin (DMD) gene leading to absent or minimal dystrophin protein

Clinical featuresProgressive muscle weakness affecting skeletal, respiratory, and cardiac muscles; usually diagnosed in early childhood (second to third year of life)

EpidemiologyPrimarily affects young males; incidence approximately 1 in 5,000 live male births in the U.S.

PrognosisProgressive decline with most patients dying of cardiac or respiratory complications in the third or fourth decade of life