Acadia Pharmaceuticals Announces DAYBUE™ (trofinetide) is Now Available for the Treatment of Rett Syndrome
Acadia Pharmaceuticals has launched DAYBUE (trofinetide), the first FDA-approved therapy for Rett syndrome, a neurodevelopmental disorder affecting 6,000 to 9,000 patients in the U.S. The drug is available for individuals aged two and older and was officially approved on March 10, 2023. The company emphasizes its commitment to patient support, expanding the Acadia Connect program to enable access to this therapy through financial assistance and prescription support. Notable endorsements from healthcare professionals highlight DAYBUE's potential to improve symptoms and enhance patients' quality of life.
Common side effects include diarrhea (experienced by 85% of patients) and weight loss (12% had losses exceeding 7%). Acadia continues to focus on treatments addressing unmet medical needs in the field of neuroscience.
- Launch of DAYBUE provides the first approved treatment for Rett syndrome, addressing a significant unmet need.
- Acadia Connect program offers comprehensive patient support, enhancing access to DAYBUE.
- The approval has garnered positive endorsements from healthcare professionals, emphasizing its potential clinical benefits.
- 85% of patients reported diarrhea, which may impact treatment adherence.
- 12% of patients experienced significant weight loss, posing a risk for discontinuation.
-- Commercial launch of DAYBUE offers Rett syndrome community the first and only approved therapy for Rett syndrome, a rare, neurodevelopmental disorder, which affects 6,000 to 9,000 patients in the
“The Rett syndrome community has been waiting a long time for a drug to treat this debilitating disorder. We have worked hard to make DAYBUE available as quickly as possible following FDA approval,” said
“Following my experience as an investigator in the Lavender Phase 3 study I have already initiated the process of prescribing DAYBUE for my patients. I am thrilled to now be able to offer DAYBUE to more people living with Rett syndrome, outside of a clinical trial,” said
Acadia Connect® Patient Access and Support Services
As part of the company’s commitment to prioritizing patient access to treatments for those who need them most, Acadia expanded the Acadia Connect® program for those prescribed DAYBUE. The multi-faceted support program offers personal assistance, financial resources and prescription support to patients and caregivers starting and continuing appropriate DAYBUE therapy. Each dedicated support team includes a nurse care coordinator, a family access manager and 24/7 clinical pharmacist support. For more information, visit AcadiaConnect.com or call 1-844-737-2223, Monday to Friday,
About Rett Syndrome
Rett syndrome is a rare, complex, neurodevelopmental disorder that may occur over four stages and affects approximately 6,000 to 9,000 patients in the
Symptoms of Rett syndrome may also include development of hand stereotypies, such as hand wringing and clapping, and gait abnormalities.8 Most Rett patients typically live into adulthood and require round-the-clock care.2,9
About DAYBUE™ (trofinetide)
Trofinetide is a synthetic version of a naturally occurring molecule known as the tripeptide glycine-proline-glutamate (GPE). The mechanism by which trofinetide exerts therapeutic effects in patients with Rett syndrome is unknown. In animal studies, trofinetide has been shown to increase branching of dendrites and synaptic plasticity signals.10,11 More information can be found at DAYBUE.com.
Important Safety Information for DAYBUE™ (trofinetide)
Important Safety Information
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Warnings and Precautions
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Diarrhea: In a 12-week study and in long-term studies, an aggregate of
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 in96% of cases. In the 12-week study, antidiarrheal medication was used in51% 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.
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Weight Loss: In the 12-week study,
12% of patients treated with DAYBUE experienced weight loss of greater than7% from baseline, compared to4% 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.
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Diarrhea: In a 12-week study and in long-term studies, an aggregate of
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Adverse Reactions: The common adverse reactions (≥
5% for DAYBUE-treated patients and at least2% greater than in placebo) reported in the 12-week study were diarrhea (82% vs20% ), vomiting (29% vs12% ), fever (9% vs4% ), seizure (9% vs6% ), anxiety (8% vs1% ), decreased appetite (8% vs2% ), fatigue (8% vs2% ), and nasopharyngitis (5% vs1% ). -
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.
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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).
Please read the accompanying full Prescribing Information, also available at DAYBUE.com
About
Acadia is advancing breakthroughs in neuroscience to elevate life. For almost 30 years we have been working at the forefront of healthcare to bring vital solutions to people who need them most. We developed and commercialized the first and only approved therapies for hallucinations and delusions associated with Parkinson’s disease psychosis and for the treatment of Rett syndrome. Our clinical-stage development efforts are focused on treating the negative symptoms of schizophrenia, Alzheimer’s disease psychosis and neuropsychiatric symptoms in central nervous system disorders. For more information, visit us at www.acadia.com and follow us on LinkedIn and Twitter.
Forward-Looking Statements
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements include but are not limited to statements regarding the timing of future events. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks and uncertainties inherent in drug development, approval and commercialization. For a discussion of these and other factors, please refer to Acadia’s annual report on Form 10-K for the year ended
References
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2 Fu C, Armstrong D, Marsh E, et al. Consensus guidelines on managing Rett syndrome across the lifespan. BMJ Paediatrics Open. 2020; 4: 1-14.
3 Kyle SM, Vashi N, Justice MJ. Rett syndrome: a neurological disorder with metabolic components. Open Biol. 2018; 8: 170216.
4
5 Amir RE, Van den Veyver IB, Wan M, et al. Rett syndrome is caused by mutations in X-linked MECP2, encoding methyl-CpG-binding protein 2.
6 Fukuda T, Itoh M, Ichikawa T, et al. Delayed maturation of neuronal architecture and synaptogenesis in cerebral cortex of Mecp2-deficient mice. J Neuropathol Exp Neurol. 2005; 64(6): 537-544.
7 Asaka Y, Jugloff DG, Zhang L, et al. Hippocampal synaptic plasticity is impaired in the Mecp2-null mouse model of Rett syndrome. Neurobiol Dis. 2006; 21(1): 217-227.
8 Neul JL, Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic criteria and nomenclature. Ann Neurol. 2010; 68(6): 944-950.
9 Daniel C, Tarquinio DO, Hou W, et al. The changing face of survival in Rett syndrome and MECP2-related disorders. Pediatr Neurol. 2015; 53(5): 402-411.
10 Tropea D, Giacometti E, Wilson NR, et al. Partial reversal of Rett Syndrome-like symptoms in MeCP2 mutant mice.
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