Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that
Health Canada has accepted its New Drug Submission (NDS) for
trofinetide for the treatment of Rett syndrome, a rare
neurodevelopmental disorder. Health Canada has granted Priority
Review for Acadia’s submission.
“Rett syndrome is a profoundly debilitating and complex
neurodevelopmental disorder that presents differently across
patients and can lead to an array of unpredictable symptoms,” said
Pamela di Cenzo, Vice President, General Manager, Rare Disease,
Canada at Acadia. “If granted marketing authorization, trofinetide
will be the first option available to treat Rett syndrome in
Canada.”
Health Canada grants Priority Review for drug submissions
intended for the treatment, prevention, or diagnosis of serious,
life-threatening, or severely debilitating illnesses or conditions
for which there is substantial evidence of clinical effectiveness
that the drug addresses an unmet medical need or provides a
benefit/risk profile that is improved over existing therapies.
“O.R.S.A. is pleased that Health Canada has granted Priority
Review for this promising treatment which, if approved, would be a
significant step forward in addressing the unmet medical needs of
Canadians living with Rett syndrome,” said Sabrina Millson,
President of the Ontario Rett Syndrome Association (O.R.S.A.). “Our
community of patients, caregivers and supporters are excited at the
prospect of having a treatment option for Rett syndrome.”
The Health Canada filing is supported by results from the
positive pivotal Phase 3 LAVENDER™ study evaluating the efficacy
and safety of trofinetide versus placebo in 187 girls and young
women 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. The key secondary
endpoint measuring improvements in communication was also
statistically significant. Trofinetide has been approved for the
treatment of Rett syndrome in adult and pediatric patients two
years of age and older in the United States, and it is not
currently authorized for sale in Canada for the treatment of Rett
syndrome.
About Rett Syndrome
Rett syndrome is a rare genetic neurodevelopmental disorder that
occurs primarily in females following a near normal development in
the first two years of life.1,2 It is caused by mutations on the X
chromosome on a gene called MECP2.3 Rett syndrome is a complex and
multisystem disorder that causes profound impairment to central
nervous system (CNS) function, including loss of communication
skills, purposeful hand use, gait abnormalities, and stereotypic
hand movements such as hand wringing/squeezing, clapping/tapping,
mouthing and washing/rubbing automatisms.2
Rett syndrome occurs worldwide in approximately one of every
10,000 to 15,000 female births.4 In Canada, prevalence of Rett
syndrome is estimated to be 600 to 900 patients.5 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.1-3 Rett syndrome is
diagnosed based on clinical evaluation, typically by about three
years of age.2,6
About Acadia Pharmaceuticals Inc.
Acadia is advancing breakthroughs in neuroscience to elevate
life. For 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 in the United States the first and
only FDA-approved drug to treat hallucinations and delusions
associated with Parkinson’s disease psychosis and the first and
only FDA-approved drug 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 Twitter.
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,” “can,” “could,” “would,”
“expects,” “plans,” “anticipates,” “believes,” “estimates,”
“projects,” “predicts,” “potential” 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 potential approval of trofinetide as the first
option in Canada for the treatment of Rett Syndrome, (ii) the
efficacy and safety profile of trofinetide for patients with Rett
Syndrome, (iii) market acceptance in Canada, including the
importance of trofinetide for the treatment of Rett Syndrome, if
approved, for Rett patients or families with patients with Rett
Syndrome and (iv) the ability for trofinetide to address the unmet
medical needs of Canadians living with Rett syndrome.
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: the ability for
trofinetide to deliver efficacious and safe results to patients, if
approved, our dependency on the continued successful
commercialization of Daybue™ in the United States, where it is
approved, our ability to obtain regulatory approval of trofinetide
in Canada and other jurisdictions outside the United States, our
ability to protect and enhance our intellectual property; 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 annual report on Form
10-K for the year ended December 31, 2023, filed with the
Securities and Exchange Commission on February 28, 2024, 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 Neul JL,
Kaufmann WE, Glaze DG, et al. Rett syndrome: revised diagnostic
criteria and nomenclature. Ann Neurol. 2010;68(6):944-950. 3 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. 4 May DM, Neul JL, Satija A, et al.
Real-world clinical management of individuals with Rett syndrome: a
physician survey. J of Med Econ. 26(1), 1570–1580. 5 Acadia
Pharmaceuticals Inc. Data on File. Canada prevalence of Rett
syndrome. February 2024. 6 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240422998763/en/
Media Contact: Acadia Pharmaceuticals Inc. Deb Kazenelson (818)
395-3043 media@acadia-pharm.com
Investor Contact: Acadia Pharmaceuticals Inc. Al Kildani (858)
261-2872 ir@acadia-pharm.com
Acadia Pharmaceuticals Inc. Jessica Tieszen (858) 261-2950
ir@acadia-pharm.com
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