CurrentUnitedHealthcarePolicy CSPA2026D0058M

Exondys 51 (eteplirsen) — Medical Benefit Drug Policy (Pennsylvania)

Defines UnitedHealthcare coverage considerations for Exondys 51 (eteplirsen) in patients with Duchenne muscular dystrophy amenable to exon 51 skipping; informs providers and payers about updated coverage criteria and related guidance.

Policy Summary

PayerUnitedHealthcare

PolicyExondys 51 (eteplirsen) — Medical Benefit Drug Policy (Pennsylvania)

Policy CodePolicy CSPA2026D0058M

Change TypeRevised coverage criteria — non-concurrency added

Effective DateJun 1, 2026

Next Review Date

Key ActionDo not authorize Exondys 51 for use concomitantly with Duvyzat (givinostat); verify plan-specific contractual requirements when making coverage decisions.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria; added criterion requiring Exondys 51 will not be used concomitantly with Duvyzat (givinostat).

Updated References section to reflect the most current information.

Archived previous policy version CSPA2025D0058L.

1non-concurrency requirement

1FDA indication noted

6references

Coverage Criteria and Rationale

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Supporting Regulatory Information

Regulatory context:

FDA indication: Exondys 51 is indicated for treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed DMD gene mutation amenable to exon 51 skipping; approval is accelerated based on increased dystrophin.

Informational; FDA approval alone is not a basis for coverage per policy text.

The document notes that FDA action is informational only: FDA approval alone is not a basis for coverage. Exondys 51’s indication and approval status are described for context, but coverage determinations must follow applicable plan terms.

When making coverage decisions, providers and payers must reference the federal, state, or contractual requirements for the benefit plan, as those requirements govern in the event of a conflict with this Medical Benefit Drug Policy. This policy is intended to assist interpretation and does not supersede the plan’s contractual terms.

Provider Responsibilities and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior authorization required. Providers must obtain prior authorization per the UnitedHealthcare Medical Benefit Drug Policy and applicable contractual requirements before initiating therapy.

Reference Medical Benefit Drug Policy for prior authorization process and supporting documentation requirements.
Verify plan-specific prior authorization forms and submission channels (portal, fax) per contractual terms.

Denial Risk

Concurrency Exclusion Risk — Duvyzat (givinostat)

Concurrent use of Exondys 51 with Duvyzat (givinostat) is excluded under the revised criteria and may result in denial of coverage. Confirm that the patient is not receiving Duvyzat prior to approving or billing for Exondys 51.

Concurrent therapy with Duvyzat (givinostat) = denial risk.
If member is on or plans to start Duvyzat, do not authorize Exondys 51 until Duvyzat use has ceased and plan-specific discontinuation documentation is provided.

Note

Plan and Contractual Verification

Providers: check and follow federal, state, and specific contract terms when applying this policy. Contractual or regulatory requirements take precedence over this standard Medical Benefit Drug Policy when conflicts exist.

Before using this policy, verify the member’s benefit plan, applicable state mandates, and any federal requirements that may alter coverage determinations.
Document the plan/contract basis when prior authorization decisions differ from this standard policy.

Documentation Required

Provider Alert — Documentation and Submission Requirements

Provider action/alert: ensure prior authorization and concurrency checks are completed before therapy initiation; include documentation that Exondys 51 will not be used concomitantly with Duvyzat when required.

Alert: Prior authorization submissions must state whether the member is taking Duvyzat and include supporting clinical documentation of absence of concurrent use.
Failure to document non-concurrency or obtain required authorization may result in claim denial or retrospective denial.

Definitions and Regulatory Status

Accelerated approval (FDA) — key points

Regulatory designationExondys 51 (eteplirsen) received FDA accelerated approval for DMD amenable to exon 51 skipping based on increased dystrophin in skeletal muscle.

Basis for approvalApproval was granted on the surrogate endpoint of dystrophin increase observed in some treated patients.

Confirmatory requirementContinued approval may be contingent on verification of clinical benefit in confirmatory trials.

Indication specificsIndicated for treatment of Duchenne muscular dystrophy in patients with a confirmed DMD gene mutation amenable to exon 51 skipping.

Coverage noteFDA approval is informational only; FDA approval alone is not a basis for coverage per policy text.