CurrentAgency for Health Care AdministrationPolicy N/A

Daybue and Daybue Stix (trofinetide) Coverage Criteria

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This policy governs prior authorization for trofinetide (Daybue and Daybue Stix) in patients with Rett syndrome. It establishes initial and continuation criteria, authorization durations, and dosing information for Medicaid fee-for-service providers.

Policy Summary

PayerAgency for Health Care Administration

PolicyDaybue and Daybue Stix (trofinetide) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage change

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization and document all initial clinical criteria before starting Daybue or Daybue Stix.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

≥2 yearsMinimum patient age

3 monthsInitial authorization

6 monthsContinuation authorization

eGFR ≥30Renal requirement

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CurrentAgency for Health Care AdministrationPolicy N/A

Daybue and Daybue Stix (trofinetide) Coverage Criteria

Customize your policy alerts

Sign up for all Agency for Health Care Administration policy alerts

Know when Agency for Health Care Administration releases new policies or updates existing guidance.

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Policy Summary

PayerAgency for Health Care Administration

PolicyDaybue and Daybue Stix (trofinetide) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage change

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization and document all initial clinical criteria before starting Daybue or Daybue Stix.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

≥2 yearsMinimum patient age

3 monthsInitial authorization

6 monthsContinuation authorization

eGFR ≥30Renal requirement

Coverage Criteria

Initial Therapy Criteria

Covered for initial therapy when all of the following are met.

Initial therapy criteria

Eligibility and diagnosis
- Age: Patient is at least 2 years of age.≥ 2 years
- Rett syndrome confirmation
  - Genetic confirmation: Molecular genetic testing confirms a heterozygous methyl-CpG binding protein 2 (MECP2) pathogenic variant gene mutation.
  - Baseline disease-severity or functionality documentation
    ONE of: Baseline disease severity of behavior and/or functionality is documented using an objective measure or tool.
    Examples include the Clinical Global Impression-Improvement score, Motor-Behavior Assessment, Interval History Form, Clinical Severity Scale, or Rett Syndrome Gross Motor Scale.
    OR: A detailed baseline clinical presentation of Rett syndrome is documented.
    The presentation may include abnormal muscle tone or dystonia, abnormal respiration pattern, feeding difficulties, intellectual disability with an IQ score below 70, loss of mobility or gait abnormalities, partial or complete loss of acquired hand skills, partial or complete loss of speech, seizures, or stereotypic hand movements.
Safety eligibility
- Renal function: Prescriber attests that the patient does not have severe renal impairment.eGFR ≥ 30 mL/min in adults or ≥ 30 mL/min/1.73 m² in pediatric patients
- Weight status: Patient does not have progressive weight loss before therapy initiation.

Continuation Therapy Criteria

Covered for continuation therapy when all of the following are met.

Continuation therapy criteria

Initial criteria: Patient met the initial review criteria.
Positive response from pretreatment baseline
- ONE of: Disease stability is documented.
- OR: Clinically significant improvement in core symptoms is documented.
- OR: An objective measure or tool demonstrates a positive response from pretreatment baseline.

Initial coverage excludes patients with severe renal impairment, defined as an estimated glomerular filtration rate (eGFR) below 30 mL/min in adults or below 30 mL/min/1.73 m² in pediatric patients.

Initial coverage excludes patients with progressive weight loss before therapy initiation.

Clinical Thresholds

Minimum age

Minimum agePatient must be at least 2 years of age.

Severe renal impairment threshold

AdultsSevere renal impairment is defined as eGFR below 30 mL/min.

Pediatric patientsSevere renal impairment is defined as eGFR below 30 mL/min/1.73 m².

Authorization and Documentation Requirements

Prior Authorization

Initial Prior Authorization Required

Prior authorization is required for Daybue® or Daybue® Stix (trofinetide). Initial authorization is limited to 3 months; a new authorization is required for continuation therapy.

Initial therapy duration: 3 months

Prior Authorization

Continuation Prior Authorization Required

Continuation prior authorization is required after the initial 3-month authorization period. Continuation authorization is limited to 6 months and requires documentation of response, treatment stability, and absence of treatment-restricting adverse effects.

Continuation therapy duration: 6 months
Submit continuation documentation before the initial authorization expires

Rett Syndrome Background

Rett syndrome is a neurodevelopmental disorder confirmed under this policy by molecular genetic testing demonstrating a heterozygous pathogenic variant in the methyl-CpG binding protein 2 (MECP2) gene, together with baseline documentation of disease severity or functionality. Clinical manifestations may affect behavior, motor function, communication, mobility, respiration, feeding, and neurologic status, including abnormal muscle tone or dystonia, abnormal respiration, feeding difficulties, intellectual disability, loss of mobility or hand skills, loss of speech, seizures, and stereotypic hand movements.

Key Definitions

MECP2 pathogenic variant

DefinitionA pathogenic variant in the methyl-CpG binding protein 2 (MECP2) gene.

Policy useMolecular genetic testing confirming a heterozygous MECP2 pathogenic variant is required to confirm the genetic basis of Rett syndrome.

Severe renal impairment

AdultsEstimated glomerular filtration rate below 30 mL/min.

Pediatric patientsEstimated glomerular filtration rate below 30 mL/min/1.73 m².

Initial Authorization Criteria

Initial Authorization

Initial authorization requires all eligibility, diagnostic, baseline documentation, and safety criteria.

Initial authorization

Patient eligibility: Patient is at least 2 years of age.≥ 2 years
Diagnostic confirmation and baseline documentation
- Genetic confirmation: Molecular genetic testing confirms a heterozygous methyl-CpG binding protein 2 (MECP2) pathogenic variant gene mutation.
- ONE of

Continuation Authorization Criteria

Continuation Authorization

Continuation authorization requires all of the following conditions.

Continuation authorization

Initial review criteria: Patient met the initial review criteria.
Positive response from pretreatment baseline
- ONE of: Disease stability is documented.
- OR: Clinically significant improvement in core symptoms is documented.

Policy Summary

PayerAgency for Health Care Administration

PolicyDaybue and Daybue Stix (trofinetide) Coverage Criteria

Policy CodePolicy N/A

Change TypeNo material clinical or coverage change

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization and document all initial clinical criteria before starting Daybue or Daybue Stix.

SourceLink

On This Page

Examples include the Clinical Global Impression-Improvement score, Motor-Behavior Assessment, Interval History Form, Clinical Severity Scale, or Rett Syndrome Gross Motor Scale.

Tolerability: Patient has not experienced treatment-restricting adverse effects.

Examples include severe diarrhea, dehydration, or significant weight loss.

Documentation Required

Initial Clinical Documentation

Initial clinical documentation must establish that the patient is eligible for therapy and has Rett syndrome confirmed by molecular genetic testing. Documentation must include a heterozygous pathogenic MECP2 variant and baseline disease severity or a detailed baseline clinical presentation.

Patient must be at least 2 years of age
Document molecular genetic testing confirming a heterozygous pathogenic MECP2 variant
Document baseline disease severity using an objective measure or tool, such as the Clinical Global Impression-Improvement (CGII) score, Motor-Behavior Assessment (MBA), Interval History Form, Clinical Severity Scale, or Rett Syndrome Gross Motor Scale; or provide a detailed baseline clinical presentation
Detailed baseline presentation may include abnormal muscle tone/dystonia, abnormal respiration, feeding difficulties, intellectual disability (IQ < 70), loss of mobility or gait abnormalities, partial or complete loss of acquired hand skills, partial or complete loss of speech, seizures, or stereotypic hand movements

Documentation Required

Renal and Weight-Loss Attestation

The prescriber must attest that the patient does not have severe renal impairment and does not have progressive weight loss before therapy is initiated.

No severe renal impairment: eGFR must not be 30 mL/min in adults or 30 mL/min/1.73 m² in pediatric patients
No progressive weight loss prior to initiation of therapy

Documentation Required

Continuation Documentation

Continuation documentation must confirm that the patient met the initial review criteria and has demonstrated a positive response from the pretreatment baseline. Response may be shown by disease stability, clinically significant improvement in core symptoms, or an objective measure or tool.

Document that the patient met initial review criteria
Document disease stability or clinically significant improvement in core symptoms
An objective measure or tool may include the CGII score, MBA, Interval History Form, Clinical Severity Scale, or Rett Syndrome Gross Motor Scale
Confirm no treatment-restricting adverse effects, including severe diarrhea or dehydration or significant weight loss

Denial Risk

Initial Eligibility Deficiencies

Initial authorization may be denied when the patient does not meet the age, genetic-confirmation, baseline-disease, or exclusion criteria required for therapy.

Patient is younger than 2 years
No documentation of molecular genetic testing confirming a heterozygous pathogenic MECP2 variant
No objective baseline disease-severity measure and no detailed baseline clinical presentation of Rett syndrome
Prescriber does not attest that severe renal impairment is absent
Progressive weight loss was present before therapy initiation

Denial Risk

Continuation Response or Safety Failure

Continuation authorization may be denied when initial eligibility is not established, the patient fails to demonstrate adequate response or stability, or treatment-restricting safety concerns are present.

Initial review criteria were not met or cannot be documented
No documented disease stability, clinically significant improvement in core symptoms, or objective evidence of response
Treatment-restricting adverse effects are present, such as severe diarrhea, dehydration, or significant weight loss

Objective baseline measure: Baseline disease severity of behavior and/or functionality is documented using an objective measure or tool.

Examples include the Clinical Global Impression-Improvement score, Motor-Behavior Assessment, Interval History Form, Clinical Severity Scale, or Rett Syndrome Gross Motor Scale.

Detailed clinical presentation: A detailed baseline clinical presentation of Rett syndrome is documented.

The presentation may include abnormal muscle tone or dystonia, abnormal respiration pattern, feeding difficulties, intellectual disability with an IQ score below 70, loss of mobility or gait abnormalities, partial or complete loss of acquired hand skills, partial or complete loss of speech, seizures, or stereotypic hand movements.

Safety criteria

Renal function: Prescriber attests that the patient does not have severe renal impairment.eGFR ≥ 30 mL/min in adults or ≥ 30 mL/min/1.73 m² in pediatric patients
Weight status: Patient does not have progressive weight loss before therapy initiation.

An objective measure or tool demonstrates a positive response from pretreatment baseline.

Examples include the Clinical Global Impression-Improvement score, Motor-Behavior Assessment, Interval History Form, Clinical Severity Scale, or Rett Syndrome Gross Motor Scale.

Adverse-effect criteria: Patient has not experienced treatment-restricting adverse effects.

Examples include severe diarrhea, dehydration, or significant weight loss.