Pharmacy Prior Authorization Criteria

Daybue (trofinetide) - PA, NF

Indications for Prior Authorization

Daybue (trofinetide)

• For diagnosis of Rett Syndrome
  Indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.

Criteria

Daybue

Prior Authorization (Initial Authorization)

Length of Approval: 3 Month(s)

• Diagnosis of Rett syndrome
AND
• One of the following:
• Presence of ALL of the following clinical signs and symptoms: [3-8]
• A pattern of development, regression, then recovery or stabilization
• Partial or complete loss of purposeful hand skills such as grasping with fingers, reaching for things, or touching things on purpose
• Partial or complete loss of spoken language
• Repetitive hand movements, such as wringing the hands, washing, squeezing, clapping, or rubbing
• Gait abnormalities, including walking on toes or with an unsteady, wide-based, stiff-legged gait
OR
• Molecular genetic testing confirms mutations in the MECP2 gene
AND
• Patient is 2 years of age or older
AND
• Prescribed by or in consultation with one of the following: [A, 2]
• Geneticist
• Neurologist

Daybue

Prior Authorization (Reauthorization)

Length of Approval: 12 Month(s)

• Patient demonstrates positive clinical response to therapy

Daybue

Non Formulary

Length of Approval: 6 Month(s)

• Submission of medical records (e.g., chart notes) confirming diagnosis of Rett syndrome
AND
• Submission of medical records (e.g., chart notes) confirming one of the following:
• Presence of ALL of the following clinical signs and symptoms: [3-8]
• A pattern of development, regression, then recovery or stabilization
• Partial or complete loss of purposeful hand skills such as grasping with fingers, reaching for things, or touching things on purpose
• Partial or complete loss of spoken language
• Repetitive hand movements, such as wringing the hands, washing, squeezing, clapping, or rubbing
• Gait abnormalities, including walking on toes or with an unsteady, wide-based, stiff-legged gait
OR
• Molecular genetic testing confirms mutations in the MECP2 gene
AND
• Patient is 2 years of age or older
AND
• Prescribed by or in consultation with one of the following: [A, 2]
• Geneticist
• Neurologist

P & T Revisions

2024-05-01, 2023-09-01, 2023-05-23, 2023-05-19

References

1. Daybue Prescribing Information. Acadia Pharmaceuticals Inc. San Diego, CA March 2023.
2. National Institute of Neurological Disorders and Stroke. Rett Syndrome. Available at: https://www.ninds.nih.gov/health-information/disorders/rett-syndrome. Accessed April 3, 2023.
3. Clinicaltrials.gov. Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDER™). Available at: https://clinicaltrials.gov/ct2/results?cond=&term=NCT04181723&cntry=&state=&city=&dist=. Accessed April 3, 2023.
4. Eunice Kennedy Shriver National Institute of Child Health and Human Development. Rett syndrome. Available at: https://www.nichd.nih.gov/health/topics/rett/conditioninfo/diagnosed. Accessed April 3, 2023.
5. Rett syndrome. Available at: https://raisingchildren.net.au/disability/guide-to-disabilities/assessment-diagnosis/rett-syndrome. Accessed April 3, 2023.
6. International Rett Syndrome Foundation. Available at: https://www.rettsyndrome.org/about-rett-syndrome/rett-syndrome-diagnosis/. Accessed April 3, 2023.
7. Neul, J., Kaufmann, W., et al. Rett Syndrome:Revised Diagnostic Criteria and Nomenclature. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3058521/. Accessed April 7, 2023.
8. Clinical consult with pediatric neurologist. April 17, 2023.
9. Optum May P & T

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End Notes

1. A neurologist, or geneticist should be consulted to confirm the diagnosis of Rett syndrome. [9]

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Revision History

• 2024-05-01: 2024 Annual Review - addition of NF criteria
• 2023-09-01: New Implementation
• 2023-05-23: update guideline
• 2023-05-19: New UM PA Criteria

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