— 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 U.S.1
SAN DIEGO–(BUSINESS WIRE)– Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that DAYBUE™ (trofinetide) is now available for the treatment of Rett syndrome in adult and pediatric patients two years of age and older in the United States. DAYBUE has demonstrated the potential to improve the signs and symptoms of Rett syndrome. DAYBUE was approved by the U.S. Food and Drug Administration (FDA) on March 10, 2023, and is the first and only drug approved by the FDA for the treatment of Rett syndrome.
“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 Steve Davis, Acadia’s Chief Executive Officer. “We are focused on providing robust patient support resources through Acadia Connect® to help caregivers and healthcare providers access this important new therapy.”
“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 Alan Percy, M.D., Professor of Pediatrics, Neurology, Neurobiology, Genetics, and Psychology at University of Alabama, Birmingham. “Having a therapy that has been shown to address multiple symptoms of Rett syndrome provides a promising treatment option that may lead to meaningful impact for patients and their families.”
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.comor call 1-844-737-2223, Monday to Friday, 8 a.m. to 8 p.m. Eastern Time.
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 U.S., with approximately 4,500 patients currently diagnosed according to an analysis of healthcare claims data.1-4 A child with Rett syndrome exhibits an early period of apparently normal development until six to 18 months, when their skills seem to slow down or stagnate. This is typically followed by a duration of regression when the child loses acquired communication skills and purposeful hand use. The child may then experience a plateau period in which they show mild recovery in cognitive interests, but body movements remain severely diminished. As they age, those living with Rett may continue to experience a stage of motor deterioration which can last the rest of the patient’s life.3 Rett syndrome is typically caused by a genetic mutation on the MECP2 gene.5 In preclinical studies, deficiency in MeCP2 function has been shown to lead to impairment in synaptic communication, and the deficits in synaptic function may be associated with Rett manifestations.5-7
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
DAYBUE is available as an oral solution (200mg/mL).
About Acadia Pharmaceuticals
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.
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 December 31, 2022, as well as Acadia’s subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are qualified in their entirety by this cautionary statement and Acadia undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law.
1 Acadia Pharmaceuticals Inc, Data on file. RTT US Prevalence. March 2022.
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 Acadia Pharmaceuticals Inc., Data on file.
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. Nat Genet. 1999; 23(2): 185-188.
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. Proc Natl Acad Sci USA. 2009; 106(6): 2029-2034.
11 Acadia Pharmaceuticals Inc., Data on file. Study Report 2566-026. 2010.
Acadia Pharmaceuticals Inc.
Acadia Pharmaceuticals Inc.
Mark Johnson, CFA
Source: Acadia Pharmaceuticals Inc.