CurrentUnitedHealthcarePolicy N/A

Exondys 51 (eteplirsen) prior authorization for Duchenne muscular dystrophy

Form and criteria governing initial and reauthorization requests for Exondys 51 (eteplirsen) for beneficiaries with Duchenne muscular dystrophy; intended for prescribing providers and UnitedHealthcare prior authorization reviewers.

Policy Summary

PayerUnitedHealthcare

PolicyExondys 51 (eteplirsen) prior authorization for Duchenne muscular dystrophy

Policy CodePolicy N/A

Change TypeNo material changes

Effective Date

Next Review Date

Key ActionComplete the prior authorization form with beneficiary, prescriber, and drug information and attach documentation confirming mutation amenable to exon 51 skipping for initial requests; include weight and dosing verification.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

initial+reauthrequest types covered by form

weight checkdosing verification

exon51 amenablemolecular eligibility

requiredprescriber signature on reauth

Coverage Criteria for Exondys 51 (eteplirsen)

Initial Authorization

Covered when ALL of the following are met for initial authorization

Initial Authorization: Beneficiary has a diagnosis of Duchenne muscular dystrophy.

Documented on prior authorization form (checkbox item 2).

Initial Authorization: Medical records confirm the DMD gene mutation is amenable to exon 51 skipping.

Attachment of supporting genetic/mutation confirmation required (checkbox item 3).

Initial Authorization: Exondys 51 is prescribed by or in consultation with a neurologist.

Prescriber relationship documented on form (checkbox item 4).

Policy Summary

PayerUnitedHealthcare

PolicyExondys 51 (eteplirsen) prior authorization for Duchenne muscular dystrophy

Policy CodePolicy N/A

Change TypeNo material changes

Effective Date

Next Review Date

SourceLink

On This Page

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Reauthorization

Reauthorization covered when AT LEAST ONE of the following is documented

Reauthorization: Documentation shows improvement in dystrophin levels.

Attach laboratory/biopsy or other objective data as indicated (form item 7).

Reauthorization: Beneficiary is not ventilator dependent.

Document functional respiratory status on reauthorization form (form item 7).

Reauthorization: Beneficiary has some functional use of upper extremities.

Clinical documentation of maintained or improved upper extremity function required (form item 7).

Reauthorization: Beneficiary has the ability to walk with or without assistive devices.

Document ambulatory status on reauthorization form (form item 7).

Prior Authorization Submission and Documentation Requirements

Prior Authorization

Prior Authorization Submission Requirements

Prior Authorization Required: Complete the beneficiary, prescriber, and drug information sections on the prior authorization form. Initial requests must include the beneficiary's current weight and documentation verifying the prescribed dosing (dose must not exceed 30 mg/kg once per week). Ensure prescriber signature and date are provided.

Include drug name, strength, quantity per 30 days, and requested length of therapy (30/60/90/120/180 days).
For initial requests: document beneficiary weight and confirm dosing ≤ 30 mg/kg once weekly.

Documentation Required

Required Documentation

Required Documentation: Attach medical records confirming the beneficiary has a diagnosis of Duchenne muscular dystrophy and that the DMD gene mutation is amenable to exon 51 skipping for initial authorization. For reauthorization, attach documentation demonstrating clinical or biomarker benefit (any of: increased dystrophin levels, not ventilator dependent, some functional use of upper extremities, or ability to ambulate with or without assistive devices).

Initial requests: medical records confirming DMD diagnosis and mutation amenable to exon 51 skipping.
Reauthorization requests: documentation of benefit — improvement in dystrophin levels, not ventilator dependent, preserved upper extremity function, or ambulatory ability.

Note

Concurrent Therapy Disclosure

Concurrent Therapy Disclosure: The form asks whether the beneficiary is taking any other RNA antisense agent or any gene therapy. All concurrent or prior RNA/gene therapies must be disclosed on the request form. Concurrent therapies may be exclusionary and will be reviewed as part of medical necessity assessment.

Answer the question on the form about other RNA antisense agents or gene therapies (Yes/No).
Failure to disclose concurrent therapies may affect coverage determination.

Denial Risk

Triggers for Possible Denial

Triggers for Possible Denial: Incomplete or missing required information may result in denial or delay. Common denial triggers include lack of documentation confirming an exon 51–amenable DMD mutation, absence of a neurologist prescriber or documented neurology consultation when required, missing beneficiary weight or dosing verification, unsigned prescriber signature, or failure to disclose concurrent RNA/gene therapies.

Missing documentation confirming amenable mutation for initial requests.
No neurologist participation or documentation of consultation when indicated.
Missing beneficiary weight, missing dosing confirmation (≤ 30 mg/kg once weekly), or unsigned prescriber signature.
Failure to attach reauthorization evidence of clinical/biomarker benefit.

OpenPayer

Exondys 51 (eteplirsen) prior authorization for Duchenne muscular dystrophy

Coverage Criteria for Exondys 51 (eteplirsen)

Dosing and Coding Constraints

Prior Authorization Submission and Documentation Requirements

Key Definitions

Background