-- DAYBUE is the first and only therapy
approved in Canada for the treatment of Rett syndrome, a rare,
neurodevelopmental disorder
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that
Health Canada has granted marketing authorization of DAYBUE™
(trofinetide) for the treatment of Rett syndrome in adult and
pediatric patients two years of age and older under the Priority
Review process. The Notice of Compliance authorization of DAYBUE
makes it the first and only drug approved in Canada for the
treatment of Rett syndrome.
Rett syndrome is a rare, complex, neurodevelopmental disorder
affecting primarily females, in which patients demonstrate
significant regression following apparently normal development in
the first two years of life.1,2 In Canada, prevalence of Rett
syndrome is estimated to be 600 to 900 patients.3 Symptoms may
include loss of communication skills, purposeful hand use, gait
abnormalities and stereotypic hand movements such as hand wringing,
clapping and rubbing.4 Most patients living with Rett syndrome will
live into adulthood and require round-the-clock care.1,5
“Health Canada’s authorization of DAYBUE is a significant
milestone for the Rett syndrome community in Canada and another
step forward in Acadia’s commitment to increase access to this
therapy for patients and their families,” said Catherine Owen
Adams, Acadia’s Chief Executive Officer. “We look forward to
bringing DAYBUE to Canadian patients with Rett syndrome as soon as
possible.”
“Rett syndrome is a debilitating and complex neurodevelopmental
disorder that presents differently across patients and can lead to
a range of symptoms throughout a patient’s life,” said Anita Datta,
MD, FRCPC, Pediatric Neurologist and Epileptologist, Co-Director of
the Rett Program at BC Children’s Hospital, and Clinical Associate
Professor at the Faculty of Medicine at UBC. “Until now, treatment
options have primarily focused on symptomatic management, as
specific therapies for Rett syndrome have not existed.”
The Health Canada filing was supported by positive results from
the pivotal Phase 3 LAVENDER™ study evaluating the efficacy and
safety of trofinetide versus placebo in 187 girls and young women
5-to-20 years of age with Rett syndrome. The co-primary endpoints
were change from baseline in the Rett Syndrome Behaviour
Questionnaire (RSBQ) total score, a caregiver assessment, and
Clinical Global Impression-Improvement (CGI-I) scale score,
clinician perspective, at week 12; both were statistically
significant. RSBQ is a 45-item rating scale completed by the
caregiver that assesses a range of symptoms of Rett syndrome
(breathing, hand movements or stereotypies, repetitive behaviors,
night-time behaviors, vocalizations, facial expressions, eye gaze,
and mood). The key secondary endpoint measuring the change from
baseline to Week 12 in the Communication and Symbolic Behavior
Scales Development Profile™ Infant-Toddler Checklist – Social
Composite Score (CSBS-DP-IT Social) was also statistically
significant when compared to placebo.6
“Rett syndrome not only has a significant impact on the
individuals living with this disorder, but on their families,
friends and loved ones as well,” said Sabrina Millson, President of
the Ontario Rett Syndrome Association (O.R.S.A.). “Today’s news is
a historic milestone for the Canadian Rett syndrome community, who
have eagerly awaited an approved treatment. Now that this therapy
has been approved, we hope it will be made accessible through
private and publicly funded drug plans as quickly as possible for
all those who could benefit from it.”
In Canada, DAYBUE is indicated for the treatment of Rett
syndrome in adults and pediatric patients two years of age and
older and weighing at least 9 kg.7
For more information, in Canada visit us at CA.Acadia.com, and
follow us on LinkedIn and X.
About Rett Syndrome
Rett syndrome is a rare, complex, neurodevelopmental disorder
that may occur over four stages and occurs in approximately one of
every 10,000 to 15,000 female births worldwide.1,2,8 In Canada,
prevalence of Rett syndrome is estimated to be 600 to 900
patients.3 Children with Rett syndrome experience a period of
developmental regression between 18-30 months of age, which is
typically followed by a plateau period lasting years to decades.2
Rett syndrome is diagnosed based on clinical evaluation, typically
by about three years of age.4,9 Rett syndrome is a complex and
multisystem disorder that causes profound impairment to central
nervous system (CNS) function, loss of communication skills,
purposeful hand use, gait abnormalities and stereotypic hand
movements such as hand wringing, clapping and rubbing.1,4 It is
caused by mutations on the X chromosome on a gene called
MECP2.10
About DAYBUE™ (trofinetide)
Trofinetide is a synthetic analog of the N-terminal tripeptide
of insulin-like growth factor 1. The mechanism by which trofinetide
exerts therapeutic effects in patients with Rett syndrome is
unknown.11
For more information, please refer to the Product Monograph for
DAYBUE in Canada located here.
About Acadia Pharmaceuticals Inc.
Acadia is advancing breakthroughs in neuroscience to elevate
life. Since our founding 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 FDA-approved
drug to treat hallucinations and delusions associated with
Parkinson’s disease psychosis and the first and only approved drug
in the United States and Canada for the treatment of Rett syndrome.
Our clinical-stage development efforts are focused on Prader-Willi
syndrome, Alzheimer’s disease psychosis and multiple other programs
targeting neuropsychiatric symptoms in central nervous system
disorders. For more information, visit us at Acadia.com and follow
us on LinkedIn and X.
Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995. Forward-looking statements include all statements other than
statements of historical fact and can be identified by terms such
as “intends,” “may,” “will,” “should,” “could,” “would,” “expects,”
“plans,” “anticipates,” “believes,” “estimates,” “projects,”
“predicts,” “potential,” “hope” and similar expressions (including
the negative thereof) intended to identify forward-looking
statements. Forward-looking statements contained in this press
release, include, but are not limited to, statements about: (i) the
efficacy and safety profile of trofinetide for patients with Rett
syndrome, (ii) market acceptance in Canada, including increased
accessibility and the importance of trofinetide for the treatment
of Rett syndrome for Rett patients or families with patients with
Rett syndrome and (iii) our estimates regarding the prevalence of
patients with Rett syndrome in Canada and worldwide.
Forward-looking statements are subject to known and unknown risks,
uncertainties, assumptions, and other factors that may cause our
actual results, performance or achievements to differ materially
and adversely from those anticipated or implied by our
forward-looking statements. Such risks, uncertainties, assumptions
and other factors include, but are not limited to: our dependency
on the commercialization of DAYBUE in Canada and the continued
successful commercialization of DAYBUE in the United States, where
it is approved, our ability to obtain regulatory approval of
trofinetide in jurisdictions outside the United States and Canada,
our ability to protect and enhance our intellectual property, risks
related to the accessibility of trofinetide to patients with Rett
syndrome through private and publicly funded drug plans and our
ability to continue to stay in compliance with applicable laws and
regulations. Given the risks and uncertainties, you should not
place undue reliance on these forward-looking statements. For a
discussion of these and other risks, uncertainties, assumptions,
and other factors that may cause our actual results, performance or
achievements to differ, please refer to our quarterly report on
Form 10-Q for the quarter ended June 30, 2024, filed with the
Securities and Exchange Commission, as well as our subsequent
filings with the Securities and Exchange Commission from time to
time. The forward-looking statements contained herein are made as
of the date hereof, and we undertake no obligation to update them
after this date, except as required by law.
References
1 Fu et al. Consensus guidelines on managing Rett syndrome
across the lifespan. BMJ Paediatrics Open. 2020;4:1-14 2 Kyle SM,
Vashi N, Justice MJ. Rett syndrome: a neurological disorder with
metabolic components. Open Biol. 2018; 8:170216. 3 Acadia
Pharmaceuticals Inc. Data on File. Canada prevalence of Rett
syndrome. September 2024. 4 Neul JL, Kaufmann WE, Glaze DG, et al.
Rett syndrome: revised diagnostic criteria and nomenclature. Ann
Neurol. 2010;68(6):944-950. 5 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. 6 Neul JL, Percy
AK, Benke TA et al. Trofinetide for the treatment of Rett syndrome:
a randomized phase 3 study. Nature Medicine. 2023; 29: 1468–1475 7
DAYBUE Canadian Product Monograph, [October] 2024 8 May DM, Neul
JL, Satija A, et al. Real-world clinical management of individuals
with Rett syndrome: a physician survey. J of Med Econ. 2023;
26(1):1570–1580 9 Tarquinio DC, Hou W, Neul JL, et al. Age of
Diagnosis in Rett Syndrome: Patterns of Recognition Among
Diagnosticians and Risk Factors for Late Diagnosis. Pediatric
Neurology. 2015;52:585-591. 10 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. 11 Acadia Pharmaceuticals Inc., Data on file.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20241016133678/en/
Investor Contact: Acadia Pharmaceuticals Inc. Al Kildani (858)
261-2872 ir@acadia-pharm.com
Media Contact: Acadia Pharmaceuticals Inc. Deb Kazenelson (818)
395-3043 media@acadia-pharm.com
Acadia Pharmaceuticals (NASDAQ:ACAD)
Graphique Historique de l'Action
De Oct 2024 à Nov 2024
Acadia Pharmaceuticals (NASDAQ:ACAD)
Graphique Historique de l'Action
De Nov 2023 à Nov 2024
