Daybue™

Drug - Daybue™ (trofinetide oral solution) [Acadia Pharmaceuticals Inc.]

March 2025

Therapeutic Area - Rett syndrome

Initial approval criteria

Patient is ≥ 2 years of age; AND
Patient has a diagnosis of classical/typical or variant/atypical Rett syndrome, as established by both of the following:
- Molecular genetic testing with heterozygous methyl-CpG-binding protein-2 (MECP2) pathogenic variant gene mutations, AND
- Clinical diagnosis based on the patient’s symptoms and behaviors; AND
Therapy will NOT be used for other genetically related (allelic) disorders; AND
Physician has assessed baseline disease severity of behavior and/or functionality using an objective measure or tool (e.g., Clinical Global Impression-Improvement [CGI-I] score, Motor-Behavior Assessment [MBA], Interval History Form, Clinical Severity Scale, Rett Syndrome Gross Motor Scale); AND
Patient does NOT have progressive weight loss prior to initiation of therapy; AND
Patient does NOT have moderate or severe renal impairment (e.g., eGFR < 45 mL/min/1.73m2)

Patient must continue to meet the above criteria; AND
Patient must have response to therapy from pre-treatment baseline with disease stability or improvement in core symptoms as evidenced on objective measure or tool (e.g., Rett Syndrome Behavior Questionnaire [RSBQ], CGI-I, MBA, Interval History Form, Clinical Severity Scale, Rett Syndrome Gross Motor scale); AND
Patient has NOT experienced any treatment-restricting adverse effects (e.g., severe diarrhea or dehydration, significant weight loss)

Max daily dose is 24 g/day (120 mL/day) to be divided into morning and evening dose
Patient’s weight must be provided at time of request

Provider Call Center (844) 575-7887