What is DAYBUE prescribed for?
DAYBUE (generic name: trofinetide), pronounced “day-BYOO,” is indicated for the treatment of Rett syndrome in adults and pediatric patients 2 years of age and older.
What is the name of the drug and what does it do?
DAYBUE is used to treat Rett syndrome in adults and children who are 2 years old or older.
Rett syndrome is a rare genetic disorder that affects brain development, causing severe physical and mental disability, mainly in girls.
It typically leads to issues with speaking, walking, eating, and breathing.
How does it work?
DAYBUE works by targeting certain aspects of Rett syndrome, a genetic disorder affecting brain development.
It helps regulate the function of specific brain pathways, potentially easing symptoms associated with the condition.
What did the research discover?
In clinical trials involving patients with Rett syndrome, DAYBUE was studied in a total of 260 patients aged 2 to 40 years, with some patients treated for varying durations: 109 for more than 6 months, 69 for more than 1 year, and 4 for more than 2 years.
The safety of DAYBUE was assessed in a 12-week study (Study 1) involving 187 female patients, mostly White, with a mean age of 11 years.
Eighteen patients (19%) discontinued DAYBUE due to adverse reactions, most commonly diarrhea (15%).
Common adverse reactions (occurring in at least 5% of patients on DAYBUE and at least 2% more frequently than with placebo) included those listed in Table 2.
Similar adverse reactions were observed in an open-label study of DAYBUE in pediatric patients aged 2 to 4 years with Rett syndrome.
DAYBUE treatment showed it worked better than placebo on key measures.
DAYBUE may interact with other medications by affecting their plasma concentrations, particularly CYP3A4 and OATP1B1/1B3 substrates, necessitating close monitoring or avoidance of concomitant use to prevent potential serious side effects.
What are some of the side effects?
The side effects listed are from the product manufacturer. As health scientists, we share this information to keep you informed without causing undue worry. Many side effects diminish over time, and not all patients experience them. If prescribed medication, take it as directed and consult your healthcare professional if you have any concerning side effects.
- Diarrhea
- Vomiting
What are the dosage recommendations and how should I take it?
The recommended dosage is twice daily, in the morning and evening, based on patient weight. DAYBUE can be taken with or without food.
Source:
DAYBUETM (trofinetide) oral solution Initial U.S. Approval: 2023 [Internet]. U.S. Food and Drug Administration. 2023 [cited 2024 Jul 1]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/217026s000lbl.pdf
