CurrentMass General Brigham Health PlanPolicy N/A

Daybue (trofinetide) — Prior Authorization for Rett Syndrome

Prior authorization policy for Daybue (trofinetide) under the pharmacy benefit for Commercial/Exchange members, including initiation and reauthorization criteria and quantity limits; specialty pharmacy dispensing required.

Policy Summary

PayerMass General Brigham Health Plan

PolicyDaybue (trofinetide) — Prior Authorization for Rett Syndrome

Policy CodePolicy N/A

Change TypeOperational / Revised documentation and quantity limits

Effective DateNov 1, 2025

Next Review DateAug 13, 2025

Key ActionObtain prior authorization and submit documentation confirming Rett syndrome with MECP2 mutation and clinical manifestations; for members new to the plan within 90 days, include proof of current receipt of therapy.

SourceLink

POLICY UPDATE CHANGES

Updated language for members who are new to the Plan and changed wording from 'medical records' to 'documentation'; added quantity limits.

≥2 yrsminimum covered age

12 monthsapproval duration

120 mL/dayquantity limit

Commercial/Exchangeplan markets

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 Daybue (trofinetide)

Initial Therapy

Covered when ALL of the following are met:

Initial Authorization for Daybue: 1. Member has a diagnosis of Rett Syndrome; 2. Documentation confirming diagnosis by a mutation in the MECP2 gene; 3. Documentation that member exhibits clinical manifestations of disease (e.g., hand wringing, apraxia, gait abnormalities, development delays); 4. Member is 2 years of age or older

Authorization may be reviewed for members new to the plan within the last 90 days who are currently receiving treatment with the requested medication, excluding samples or manufacturer patient assistance programs.

Continuation Therapy

Covered when ALL of the following are met:

Continuation/Reauthorization: Member is experiencing benefit from therapy (e.g., stabilization or improvement in repetitive movements, mood dysfunction/disruptive behavior, vocalization, ambulation).

Reauthorizations and initial approvals are granted for 12 months.

For members who are new to the plan within the past 90 days and are currently receiving Daybue, submit documentation confirming current receipt of the medication to be eligible for prior authorization. Documentation should exclude drug samples and manufacturer's patient assistance program supplies. Requests lacking this documentation may be subject to review or denial if ongoing therapy cannot be verified.

Daybue is not covered when used in patients who are younger than 2 years of age. Additionally, therapy is not covered in the absence of a documented pathogenic MECP2 mutation and accompanying clinical manifestations of Rett syndrome (for example, hand wringing, apraxia, gait abnormalities, or developmental delays).

Coding and Quantity Limits

Prior authorization affected codesmixed

affected codes

Placeholder — document lists 'affected codes' for prior auth but does not enumerate specific billing or NDC codes.

Quantity limit for Daybue oral solution

Quantity limit120 mL per day

DrugDaybue oral solution

Approval duration associatedInitial and reauthorizations granted for 12 months

Provider Actions, Documentation, and Denial Triggers

Prior Authorization

Prior Authorization Required

Prior authorization is required for Daybue (trofinetide) under the pharmacy benefit for Commercial/Exchange members. This drug is designated specialty and must be dispensed by a contracted specialty pharmacy; submit prior authorization requests to the pharmacy benefit contact.

Applies to Commercial/Exchange members under the pharmacy benefit
Specialty dispensing required — must be filled at a contracted specialty pharmacy
Pharmacy benefit PA contact: Phone: 800-711-4555; Fax: 844-403-1029

Step Therapy

No Step Therapy Requirement

No step therapy (prior failed-agent) requirement is specified for Daybue in this policy.

Background

Rett syndrome is a neurodevelopmental disorder caused by mutations in the MECP2 gene and is characterized by developmental regression and features such as repetitive hand stereotypies (eg, hand wringing), loss of purposeful hand use, apraxia, gait abnormalities, and communication impairments. Daybue (trofinetide) is indicated for treatment of Rett syndrome in patients aged 2 years and older.

Definitions

Rett syndrome (MECP2-confirmed)

DefinitionRett syndrome diagnosed by a pathogenic mutation in the MECP2 gene with clinical manifestations

Required diagnostic confirmationDocumentation confirming diagnosis by a mutation in the MECP2 gene

Minimum age for therapy2 years of age or older

Typical clinical manifestationsHand wringing (stereotypies), apraxia, gait abnormalities, developmental delays