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The first and only
FDA-approved treatment
for Rett syndrome





ADD MORE OF HER
SPARKLE TO THE WORLD AROUND THEM

DAYBUE™ (trofinetide) is the first and only FDA-approved treatment for Rett
syndrome (RTT) in adults and pediatric patients 2 years of age and older.1,2

DAYBUE provides an opportunity to help spark meaningful improvements in the
signs and symptoms of RTT.*

Learn more about the efficacy data. >

Kate, age 9, living with
Rett syndrome (left) and
her parents (right)

DAYBUE™ (trofinetide) is the first and only FDA-approved treatment for Rett
syndrome (RTT) in adults and pediatric patients 2 years of age and older.1,2

DAYBUE provides an opportunity to help spark meaningful improvements in the
signs and symptoms of RTT.*

 

Learn more about the efficacy data. >


EXPLORE HOW DAYBUE WAS STUDIED

Review LAVENDER™, the pivotal Phase 3 trial.1,3

See the study design


SEE PROVEN EFFICACY DATA1

View results of changes in RSBQ total score and CGI-I score from baseline to
Week 12.

Explore the data


REVIEW SAFETY AND TOLERABILITY PROFILE1

Review side effect information, including safety and tolerability data and
potential management strategies.

View the safety profile

*Improvement in signs and symptoms of RTT (breathing, hand movements or
stereotypies, repetitive behaviors, night-time behaviors, vocalizations, facial
expressions, eye gaze, and mood) were evaluated in a 12-week study as measured
by the mean change from baseline in RSBQ total score and CGI-I score vs
placebo.1

Kate, age 9, living with Rett Syndrome, with her brother.


QUESTIONS ABOUT DAYBUE?

Our representatives are here with information on this first and only treatment
for RTT.

Request a rep

Learn about the symptoms of RTT and the impact they can have on individuals and
their caregivers.

About Rett syndrome >


CGI-I=Clinical Global Impression-Improvement; RSBQ=Rett Syndrome Behaviour
Questionnaire.


IMPORTANT SAFETY INFORMATION AND INDICATION

 * Warnings and Precautions
   
   * Diarrhea: In a 12-week study and in long-term studies, 85% of patients
     treated with DAYBUE experienced diarrhea. In those treated with DAYBUE, 49%
     either had persistent diarrhea or recurrence after resolution despite dose
     interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea
     severity was of mild or moderate severity in 96% of cases. In the 12-week
     study, antidiarrheal medication was used in 51% of patients treated with
     DAYBUE.
     
     Patients should stop taking laxatives before starting DAYBUE. If diarrhea
     occurs, patients should notify their healthcare provider, consider starting
     antidiarrheal treatment, and monitor hydration status and increase oral
     fluids, if needed. Interrupt, reduce dose, or discontinue DAYBUE if severe
     diarrhea occurs or if dehydration is suspected.
   
   * Weight Loss: In the 12-week study, 12% of patients treated with DAYBUE
     experienced weight loss of greater than 7% from baseline, compared to 4% of
     patients who received placebo. In long-term studies, 2.2% of patients
     discontinued treatment with DAYBUE due to weight loss. Monitor weight and
     interrupt, reduce dose, or discontinue DAYBUE if significant weight loss
     occurs.

 * Adverse Reactions: The common adverse reactions (≥5% for DAYBUE-treated
   patients and at least 2% greater than in placebo) reported in the 12-week
   study were diarrhea (82% vs 20%), vomiting (29% vs 12%), fever (9% vs 4%),
   seizure (9% vs 6%), anxiety (8% vs 1%), decreased appetite (8% vs 2%),
   fatigue (8% vs 2%), and nasopharyngitis (5% vs 1%).

 * Drug Interactions: Effect of DAYBUE on other Drugs
   
   * DAYBUE is a weak CYP3A4 inhibitor; therefore, plasma concentrations of
     CYP3A4 substrates may be increased if given concomitantly with DAYBUE.
     Closely monitor when DAYBUE is used in combination with orally administered
     CYP3A4 sensitive substrates for which a small change in substrate plasma
     concentration may lead to serious toxicities.
   
   * Plasma concentrations of OATP1B1 and OATP1B3 substrates may be increased if
     given concomitantly with DAYBUE. Avoid the concomitant use of DAYBUE with
     OATP1B1 and OATP1B3 substrates for which a small change in substrate plasma
     concentration may lead to serious toxicities.

 * Use in Specific Population: Renal Impairment
   
   * DAYBUE is not recommended for patients with moderate or severe renal
     impairment.

DAYBUE is available as an oral solution (200mg/mL).

 * Indication

DAYBUE is indicated for the treatment of Rett syndrome in adults and pediatric
patients 2 years of age and older.

Please read the full Prescribing Information.


IMPORTANT SAFETY INFORMATION AND INDICATION

See more


 * Warnings and Precautions
   
   * Diarrhea: In a 12-week study and in long-term studies, 85% of patients
     treated with DAYBUE experienced diarrhea. In those treated with DAYBUE, 49%
     either had persistent diarrhea or recurrence after resolution despite dose
     interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea
     severity was of mild or moderate severity in 96% of cases. In the 12-week
     study, antidiarrheal medication was used in 51% of patients treated with
     DAYBUE.
     
     Patients should stop taking laxatives before starting DAYBUE. If diarrhea
     occurs, patients should notify their healthcare provider, consider starting
     antidiarrheal treatment, and monitor hydration status and increase oral
     fluids, if needed. Interrupt, reduce dose, or discontinue DAYBUE if severe
     diarrhea occurs or if dehydration is suspected.
   
   * Weight Loss: In the 12-week study, 12% of patients treated with DAYBUE
     experienced weight loss of greater than 7% from baseline, compared to 4% of
     patients who received placebo. In long-term studies, 2.2% of patients
     discontinued treatment with DAYBUE due to weight loss. Monitor weight and
     interrupt, reduce dose, or discontinue DAYBUE if significant weight loss
     occurs.

 * Adverse Reactions: The common adverse reactions (≥5% for DAYBUE-treated
   patients and at least 2% greater than in placebo) reported in the 12-week
   study were diarrhea (82% vs 20%), vomiting (29% vs 12%), fever (9% vs 4%),
   seizure (9% vs 6%), anxiety (8% vs 1%), decreased appetite (8% vs 2%),
   fatigue (8% vs 2%), and nasopharyngitis (5% vs 1%).

 * Drug Interactions: Effect of DAYBUE on other Drugs
   
   * DAYBUE is a weak CYP3A4 inhibitor; therefore, plasma concentrations of
     CYP3A4 substrates may be increased if given concomitantly with DAYBUE.
     Closely monitor when DAYBUE is used in combination with orally administered
     CYP3A4 sensitive substrates for which a small change in substrate plasma
     concentration may lead to serious toxicities.
   
   * Plasma concentrations of OATP1B1 and OATP1B3 substrates may be increased if
     given concomitantly with DAYBUE. Avoid the concomitant use of DAYBUE with
     OATP1B1 and OATP1B3 substrates for which a small change in substrate plasma
     concentration may lead to serious toxicities.

 * Use in Specific Population: Renal Impairment
   
   * DAYBUE is not recommended for patients with moderate or severe renal
     impairment.

DAYBUE is available as an oral solution (200mg/mL).

 * Indication

DAYBUE is indicated for the treatment of Rett syndrome in adults and pediatric
patients 2 years of age and older.

Please read the full Prescribing Information.

References: 1. Acadia Pharmaceuticals Inc. DAYBUE [Package Insert]. San Diego,
CA, 2023. 2. Acadia Pharmaceuticals announces U.S. FDA approval of DAYBUE™
(trofinetide) for the treatment of Rett syndrome in adult and pediatric patients
two years of age and older. [press release]. Acadia Pharmaceuticals Inc. March
10, 2023. 3. Neul JL, Percy AK, Benke TA, et al. Design and outcome measures of
LAVENDER, a phase 3 study of trofinetide for Rett syndrome. Contemp Clin Trials.
2022;114:106704.

This website is intended for use by US healthcare professionals.

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©2023 Acadia Pharmaceuticals Inc.
Acadia and Acadia Connect are registered trademarks and DAYBUE is a trademark of
Acadia Pharmaceuticals Inc. All other trademarks are the property of their
respective owners. All rights reserved.
DAY-0073-v3 11/23

Acadia is trailblazing breakthroughs in neuroscience to elevate life. For more
than 25 years, we have been working at the forefront of healthcare to bring
vital solutions to people who need them most.
12830 El Camino Real, Suite 400
San Diego, CA 92130

Acadia is trailblazing breakthroughs in neuroscience to elevate life. For more
than 25 years, we have been working at the forefront of healthcare to bring
vital solutions to people who need them most.
12830 El Camino Real, Suite 400
San Diego, CA 92130

©2023 Acadia Pharmaceuticals Inc.
Acadia and Acadia Connect are registered trademarks and DAYBUE is a trademark of
Acadia Pharmaceuticals Inc. All other trademarks are the property of their
respective owners. All rights reserved.
DAY-0073-v3 11/23


WELCOME TO THE DAYBUE™ (TROFINETIDE) WEBSITE.

This information is intended for US healthcare professionals only.

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USING THE RSBQ, CAREGIVERS WERE ASKED TO SCORE 45 SYMPTOMS OF RTT ON A SCALE
FROM 0 (NOT TRUE) TO 2 (VERY/OFTEN TRUE).1 THE RATING SCALE AND ITEMS IN THE
RSBQ ARE SHOWN BELOW.9

 * Hand movements are uniform and monotonous

 * Has difficulty in breaking/stopping hand stereotypies

 * There are times when breathing is deep and fast (hyperventilation)

 * There are times when breath is held

 * The amount of time spent looking at objects is longer than the time spent
   holding or manipulating them

 * Makes mouth grimaces

 * There are times when parts of the body are held rigid

 * Restricted repertoire of hand movements

 * Has wounds on hands as a result of repetitive hand movements

 * Makes grimacing expressions with face

 * Swallows air

 * Does not use hands for purposeful grasping

 * Tendency to bring hands together in front of chin or chest

 * Air or saliva is expelled from mouth with force

 * Seems frightened when there are sudden changes in own body position

 * Spells of laughter for no apparent reason during the day

 * Grinds teeth

 * Uses eye gaze to convey feelings, needs, and wishes

 * Shifts gaze with a slow horizontal turn of head

 * Spells of inconsolable crying for no apparent reason during the day

 * Spells of laughter for no apparent reason during the night

 * Spells of apparent panic

 * Has frequent naps during the day

 * Spells of screaming for no apparent reason during the day

 * Makes repetitive hand movements with hands apart

 * Screams hysterically for long periods of time and cannot be consoled

 * Spells of screaming for no apparent reason during the night

 * Vocalizes for no apparent reason

 * Rocks self when hands are prevented from moving

 * There are times when she appears miserable for no apparent reason

 * Abrupt changes in mood

 * Spells of inconsolable crying for no apparent reason during the night

 * Makes repetitive movements involving fingers around tongue

 * Rocks body repeatedly

 * Appears isolated

 * Vacant “staring” spells

 * Abdomen fills with air and sometimes feels hard

 * There are certain periods where she performs much worse than usual

 * There are times when she is irritable for no apparent reason

 * Seems to look through people into the distance

 * Although can stand independently tends to lean on objects or people

 * Expressionless face

 * Spells of apparent anxiety/fear in unfamiliar situations

 * Makes repetitive tongue movements

 * Walks with stiff legs




SELECT INCLUSION CRITERIA6

 * Female subjects 5 to 20 years of age

 * Body weight ≥12 kg at screening

 * Could swallow the study medication provided as a liquid solution or could
   take it by gastrostomy tube

 * Had classic/typical RTT

 * Had a documented disease-causing mutation in the MECP2 gene

 * Had a stable pattern of seizures, or had no seizures within 8 weeks of
   screening



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