Currentmedical mutual - ohioPolicy N/A

Daybue (trofinetide)

Pharmacy benefit policy describing prior authorization recommendation, required prescriber specialty/consultation, approval expectations, documentation rights, and conditions not recommended for approval for Daybue (trofinetide) for treatment of Rett syndrome in patients 6 2 years.

Policy Summary

Payermedical mutual - ohio

PolicyDaybue (trofinetide)

Policy CodePolicy N/A

Change TypeAnnual reviewno material change

Effective Date

Next Review DateAug 21, 2025

Key ActionSubmit prior authorization requests for Daybue and ensure initial prescriptions are by or in consultation with a specialist; provide response documentation for reauthorization.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes for Daybue (trofinetide) — has_material_change=false.

1Indication specified

0Recommended Authorization Criteria items

3Key clinical trials cited

Coverage Summary & Stance

Coverage stance: mixed. Daybue (trofinetide) is indicated for treatment of Rett syndrome in adults and pediatric patients aged ≥ 2 years.

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Recommended Authorization Criteria

None. Note: The brief provided no detailed recommended authorization criteria; include this intro and an explicit note that no specific authorization criteria were specified in the brief/policy.

Recommended Authorization Criteria: None specified in the source policy. This group is included to hold future initial-therapy criteria; the brief/policy explicitly states no recommended authorization criteria were provided.

No detailed criteria were specified in the brief (see intro).

Conditions Not Recommended for Approval

Daybue is not recommended for general approval for the following conditions (not exhaustive):

Conditions Not Recommended for Approval: Rett syndrome: efficacy evaluated in trials (LAVENDER pivotal, RETT-002 dose-ranging, DAFFODIL PK bridging); evidence shows marginal efficacy on subjective co-primary endpoints and was limited to female patients with typical Rett syndrome and documented MECP2 mutation post-regression ≥ 6 months at screening.Post-regression status ≥ 6 months

Coverage is not recommended for circumstances not listed in the Recommended Authorization Criteria; criteria will be updated as new published data are available.

Authorization & Prescriber Requirements

Authorization and Prescriber Requirements

Policy statements regarding authorization and prescriber:

Authorization and Prescriber Requirements: Prior authorization is recommended for pharmacy benefit coverage of Daybue.

Authorization and Prescriber Requirements: Initial approval requires Daybue be prescribed by or in consultation with a physician who specializes in the condition being treated due to specialized skills required for evaluation, diagnosis, and monitoring.

Documentation of specialist involvement may be requested.

Authorization and Prescriber Requirements: All approvals for initial therapy are provided for the initial approval duration noted; if reauthorization is allowed, a response to therapy is required for continuation of therapy unless otherwise noted.

Provider Actions & Documentation Requirements

Prior Authorization

Prior authorization recommended

Submit a prior authorization request for Daybue. Requests for uses not listed in this policy will be reviewed case-by-case for evidence of efficacy and medical necessity.

Documentation Required

Specialist prescriber or consultation required for initial approval

Initial prescriptions should be by or in consultation with a physician who specializes in Rett syndrome or the treated condition. Documentation of specialist involvement may be requested as part of the coverage determination.

Documentation Required

Response required for reauthorization

If reauthorization is allowed, provide clinical documentation demonstrating response to therapy for continuation of Daybue.

Documentation Required

Company may request additional documentation

The Company may request patient records, test results, provider credentials, or other documentation to support medical necessity. Lack of medical necessity or use considered investigational may result in denial of reimbursement.

Clinical Evidence & Trials

3Key clinical trials cited

12 wksPivotal trial duration (LAVENDER)

MarginalPivotal trial outcome on co-primary endpoints (RSBQ, CGI-I)

Evidence summary: Key clinical trials referenced include the phase 3 LAVENDER pivotal trial, the RETT-002 dose-ranging trial, and the DAFFODIL pharmacokinetic bridging study.

LAVENDER demonstrated marginal efficacy on the subjective co-primary endpoints (RSBQ and CGI-I) at 12 weeks.

Background & Definitions

Daybue (trofinetide) is indicated for the treatment of Rett syndrome in adults and pediatric patients aged ≥ 2 years.

Rett syndrome is a neurodevelopmental disorder typically caused by mutations in the MECP2 gene. Symptoms usually appear between 6 to 18 months of age and include loss of developmental milestones, loss of hand function and distinctive hand movements, slowed head growth, problems with walking, seizures, and intellectual disability.

The product labeling and policy note an age threshold of ≥ 2 years for the indication; supportive PK bridging data for patients 2 to 4 years were provided by the DAFFODIL study.

Post-regression status: defined as no loss or degradation in ambulation, hand function, speech, nonverbal communicative or social skills for ≥ 6 months at screening. See Coverage section for the ≥ 6 months post-regression threshold used in trial enrollment criteria.

Policy Summary

Payermedical mutual - ohio

PolicyDaybue (trofinetide)

Policy CodePolicy N/A

Change TypeAnnual reviewno material change

Effective Date

Next Review DateAug 21, 2025

Key ActionSubmit prior authorization requests for Daybue and ensure initial prescriptions are by or in consultation with a specialist; provide response documentation for reauthorization.

SourceLink

On This Page

OpenPayer

Daybue (trofinetide)

Coverage Summary & Stance

Recommended Authorization Criteria

Conditions Not Recommended for Approval

Authorization & Prescriber Requirements

Provider Actions & Documentation Requirements

Applicable Codes

Clinical Evidence & Trials

Background & Definitions

Revision History