DAYBUE (trofinetide)
Self-Administration – oral
Diagnosis considered for coverage:
- Rett Syndrome: Indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.
Coverage Criteria:
- Diagnosis of Rett syndrome, AND
- Patient is 2 years of age or older, AND
- Patient’s current weight is provided, AND
- Prescribed by or in consultation with a geneticist or neurologist, AND
- One of the following:
- Presence of ALL of the following clinical signs and symptoms:
- 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
- Presence of ALL of the following clinical signs and symptoms:
Reauthorization Criteria:
- Documentation of positive clinical response to therapy
Dosing:
- DAYBUE is administered orally twice daily, in the morning and evening, according to patient weight as shown in the table below:
|
Patient Weight |
DAYBUE Dosage |
DAYBUE Volume |
|
9 kg to less than 12 kg |
5,000 mg twice daily |
25 mL twice daily |
|
12 kg to less than 20 kg |
6,000 mg twice daily |
30 mL twice daily |
|
20 kg to less than 35 kg |
8,000 mg twice daily |
40 mL twice daily |
|
35 kg to less than 50 kg |
10,000 mg twice daily |
50 mL twice daily |
|
50 kg or more |
12,000 mg twice daily |
60 mL twice daily |
Coverage Duration:
- Initial: 3 months
- Reauthorization: 12 months
Authorization is not covered for the following:
The use of this drug for indications not listed in this policy does not meet the coverage criteria established by the Western Health Advantage (WHA) Pharmacy and Therapeutics (P&T) Committee.
Additional Information:
- A neurologist, or geneticist should be consulted to confirm the diagnosis of Rett syndrome.
- There is no cure for RTT; treatment focuses on symptom management from a multidisciplinary team
- Because trofinetide is eliminated mainly through the kidneys, administration to patients with moderate or severe renal impairment is not recommended.
Policy Updates:
- 08/15/2023 – New policy approved by P&T.
References:
- Daybue Prescribing Information. Acadia Pharmaceuticals Inc. San Diego, CA March 2023.
- 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.
- 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.
- 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.
- Rett syndrome. Available at: https://raisingchildren.net.au/disability/guide-to-disabilities/assessment-diagnosis/rett-syndrome. Accessed April 3, 2023.
- International Rett Syndrome Foundation. Available at: https://www.rettsyndrome.org/about-rett-syndrome/rett-syndrome-diagnosis/. Accessed April 3, 2023.
- 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.
- Clinical consult with pediatric neurologist. April 17, 2023.
- Optum May P & T
Last review date: September 1, 2023