Acadia Pharmaceuticals Announces DAYBUE™ (trofinetide) is Now Available for the Treatment of Rett Syndrome
— 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
- 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 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).
Please read the accompanying full Prescribing Information, also available at DAYBUE.com
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.
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 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.
References
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.
View source version on businesswire.comhttps://www.businesswire.com/news/home/20230417005353/en/
Contacts
Media Contact:
Acadia Pharmaceuticals Inc.
Deb Kazenelson
(818) 395-3043
media@acadia-pharm.com
Investor Contact:
Acadia Pharmaceuticals Inc.
Mark Johnson, CFA
(858) 261-2771
ir@acadia-pharm.com
Source: Acadia Pharmaceuticals Inc.
DayBue Announcement
Last Updated: 07/26/2023 by admin
Acadia Pharmaceuticals Announces DAYBUE™ (trofinetide) is Now Available for the Treatment of Rett Syndrome
— 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
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.
DAYBUE is available as an oral solution (200mg/mL).
Please read the accompanying full Prescribing Information, also available at DAYBUE.com
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.
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 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.
References
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.
View source version on businesswire.comhttps://www.businesswire.com/news/home/20230417005353/en/
Contacts
Media Contact:
Acadia Pharmaceuticals Inc.
Deb Kazenelson
(818) 395-3043
media@acadia-pharm.com
Investor Contact:
Acadia Pharmaceuticals Inc.
Mark Johnson, CFA
(858) 261-2771
ir@acadia-pharm.com
Source: Acadia Pharmaceuticals Inc.
Neurogene Announcement
Last Updated: 07/26/2023 by admin
DAYBUE in Canada
Last Updated: 07/26/2023 by Steve
Is DAYBUE approved outside of the US?
At this time, DAYBUE is only approved in the US for the treatment of Rett syndrome in adults and children 2 years of age and older. If you have a child who is living with Rett syndrome, we understand your urgency to learn what possible treatments may be available for your child. While Acadia Pharmaceuticals Inc. owns the rights to trofinetide in North America (Canada, Mexico and the US), Neuren Pharmaceuticals owns the rights outside of North America.
If you live in the US: DAYBUE was FDA approved on March 10, 2023. By the end of April 2023, DAYBUE will be available by prescription through your doctor. Once you receive your child’s prescription for DAYBUE, you’ll be connected to the dedicated support team at Acadia Connect. The Acadia Connect team will be at your side every step of the way to support you in starting treatment with DAYBUE. You can reach Acadia Connect at 1-844-737-2223.
If you live in Canada: With the US approval complete, we are in the midst of developing a regulatory and commercial plan to commercialize trofinetide in Canada, and will provide further details on the timeline as we progress. For any additional inquiries, please do not hesitate to contact Acadia Medical Information at 1-844-4ACADIA (1-844-422-2342) Option 1 or medicalinformation@acadia-pharm.com
daybue-faqCHEO Clinic Update
Last Updated: 06/06/2023 by Steve
18th Annual Rett Classic – Sunday June 11, 2023
Last Updated: 06/06/2023 by Steve
We are excited to announce the date for our
18th Annual Rett Classic
Join us for golf, dinner, & friendship in support of O.R.S.A.
Sunday, June 11, 2023 at our NEW location in
Flamborough, ON.
Register Today!
https://www.canadahelps.org/en/charities/ontario-rett-syndrome-association/events/rett-classic/
Last Updated: 02/16/2024 by admin
CANADIAN RETT SYNDROME REGISTRY
Dear National Registry Users,
We are thrilled to announce that the Canadian Rett Syndrome National Registry recently underwent a temporary offline period for a significant upgrade aimed at securing your data and providing enhanced customization options to better serve your needs. This upgrade comes just in time for our 10-year anniversary, marking a decade of commitment to excellence.
Key Enhancements:
New Options Available:
We appreciate your patience during the temporary downtime and hope you enjoy the enhanced features.
Thank you for being a valued part of the Canadian Rett Syndrome National Registry community.
Taysha Rett Community Letter
Last Updated: 07/26/2023 by Steve
Taysha-Rett-Community-Letter-FEB-1-2023
2022 Year in Review
Last Updated: 06/06/2023 by Steve
2022 Run4Rett
Last Updated: 06/16/2023 by Steve
The month of October encompassed Rett syndrome awareness and the 2022 RUN4RETT virtually. Families and friends could participate in their own backyard. Connected together with the unbreakable spirit of all our loved ones with Rett syndrome.
The event registration, for over 10 years, has been found at the Running Room site. Thank you for believing we can make a difference, with every participant, registration and donation made.
Thank you to the Swartz and Dodds families. Your support and generous donations of auction items every year has been unwavering. O.R.S.A. has been able to count on you since the beginning. Once again we had a student volunteer assisting with the auction photos. Thank you Tyler Polon.
Together you are making a difference to those affected with Rett syndrome and their families.
The success of the 2022 virtual event and silent auction speaks volumes of the support from the families and friends of O.R.S.A. The total combined money raised was just over $39,000. Thank you to all those that participated, registered, donated and bid. Together we are making a difference.
Mark your calendars, September 24th, 2023 at a new location, the Richvale CC at 160 Avenue Road, Richmond Hill, Ontario, see you there!
Support O.R.S.A. by using FlipGive
Last Updated: 06/06/2023 by Steve
You can support O.R.S.A. by purchasing the items you already need or want. Here’s how:
Join our team now using our invite code SP69V5 in one of 2 ways:
1. Download the FlipGive app at Click Here and enter team code: SP69V5
2. Join online Click Here