-- NDA granted priority review
-- Prescription Drug User Fee Act action date
set for March 12, 2023
Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that
the U.S. Food and Drug Administration (FDA) has accepted for filing
its New Drug Application (NDA) of trofinetide for the treatment of
Rett syndrome. The FDA has granted a priority review and assigned a
PDUFA (Prescription Drug User Fee Act) action date of March 12,
2023. The FDA has also informed the company that at this time they
are not planning to hold an Advisory Committee meeting.
“We’re pleased that the FDA has accepted our NDA filing and we
will be working closely with them to facilitate completion of the
review in a timely manner,” said Steve Davis, Acadia’s Chief
Executive Officer. “If approved, trofinetide will be the first drug
available for the treatment of Rett syndrome, a rare and
devastating condition for patients and their families. This
milestone reinforces Acadia’s ongoing commitment to advancing
research into high unmet needs in disorders affecting the central
nervous system.”
Rett syndrome is a complex, multisystem neurodevelopmental
disorder that includes a period of normal development followed by
significant developmental regression with loss of language and hand
function skills, impaired gait and development of hand
stereotypes.1,2 It occurs worldwide in approximately one of every
10,000 to 15,000 female births.3
“Rett is a complex disease that can present with a diverse array
of symptoms. In clinical trials, trofinetide demonstrated a
significant improvement in a range of Rett syndrome symptoms,” said
Jeffrey L. Neul, M.D., Ph.D., Annette Schaffer Eskind Chair and
Director, Vanderbilt Kennedy Center, Professor of Pediatrics,
Division of Neurology, Pharmacology, and Special Education,
Vanderbilt University Medical Center and Phase 3 Lavender™ study
investigator. “We look forward to the FDA’s review of this
submission and the prospect of having access to the first approved
treatment for Rett syndrome.”
The NDA is supported by results from the pivotal Phase 3
Lavender study evaluating the efficacy and safety of trofinetide
versus placebo in 187 girls and young women aged 5-20 years with
Rett syndrome. The study demonstrated a statistically significant
improvement over placebo on the co-primary endpoints, the Rett
Syndrome Behaviour Questionnaire (RSBQ) total score change from
baseline to 12 weeks (p=0.0175; effect size=0.37) and the Clinical
Global Impression-Improvement (CGI-I) scale score (p=0.0030; effect
size=0.47). The RSBQ is a caregiver assessment of the core symptoms
of Rett syndrome, and the CGI-I is a global physician assessment of
worsening or improving of Rett syndrome. In addition, the study
also met its key secondary endpoint, the Communication and Symbolic
Behavior Scales Developmental Profile™ Infant-Toddler
Checklist–Social Composite Score (CSBS-DP-IT–Social) change from
baseline to week 12 (p=0.0064; effect size=0.43), a caregiver
assessment of ability to communicate.
In 2018, Acadia entered into an exclusive license agreement with
Neuren Pharmaceuticals Limited (ASX: NEU) for the development and
commercialization of trofinetide for the treatment of Rett syndrome
and other indications in North America. In addition to receiving
priority review by the FDA, trofinetide has been granted Fast Track
Status and Orphan Drug Designation for the treatment of Rett
syndrome in the U.S. and has been granted Rare Pediatric Disease
(RPD) designation by the FDA. Upon FDA approval of a product with
RPD designation, the sponsor can receive a Priority Review Voucher,
which can be used to obtain priority review for a subsequent
application.
About Lavender™ The Lavender study was a Phase 3, 12-week,
double-blind, randomized, placebo-controlled study of trofinetide
in 187 girls and young women aged 5-20 years with Rett syndrome,
designed to evaluate its efficacy and safety. The co-primary
endpoints of Lavender included both a caregiver (Rett Syndrome
Behaviour Questionnaire [RSBQ]) and physician (Clinical Global
Impression–Improvement [CGI-I]) assessment. The key secondary
endpoint was also a caregiver assessment designed to evaluate
non-verbal communication skills, the Communication and Symbolic
Behavior Scales Developmental Profile™ Infant‑Toddler Checklist –
Social Composite Score (CSBS-DP- IT–Social).
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 MECP2.4 Occurring worldwide in approximately one of every
10,000 to 15,000 female births and in the United States impacts
6,000 to 9,000 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.1,2,4 Rett syndrome is diagnosed based on clinical
evaluation, typically by about three years of age.2,5
A complex and multisystem disorder, Rett syndrome 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 People living with Rett syndrome may also experience
a range of additional symptoms, such as gastrointestinal
complications, skeletal abnormalities, neuroendocrine
abnormalities, disruptive and anxiety-like behaviors, as well as
mood dysregulation and sleep disturbances.1 Currently, there are no
FDA-approved medicines for the treatment of Rett syndrome.
About Trofinetide Trofinetide is an investigational drug. It is
a novel synthetic analog of the amino‐terminal tripeptide of IGF-1
designed to treat the core symptoms of Rett syndrome by potentially
reducing neuroinflammation and supporting synaptic function.
Trofinetide is thought to stimulate synaptic maturation and
overcome the synaptic and neuronal immaturities that are
characteristic of Rett syndrome pathophysiology. In the central
nervous system, IGF-1 is produced by both of the major types of
brain cells – neurons and glia. IGF-1 in the brain is critical for
both normal development and for response to injury and disease.
Trofinetide has been shown to inhibit the production of
inflammatory cytokines, inhibit the overactivation of microglia and
astrocytes, and increase the amount of available IGF-1 that can
bind to IGF-1 receptors.
About Acadia Pharmaceuticals Acadia is advancing breakthroughs
in neuroscience to elevate life. For more than 25 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 therapy for
hallucinations and delusions associated with Parkinson’s disease
psychosis. Our clinical-stage development efforts are focused on
treating the negative symptoms of schizophrenia, Rett syndrome 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, 2021, 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 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 U.S.
prevalence estimate based on incidence rates from the National
Institutes of Health – National Institute of Neurological Disorders
and Stroke. 4 Amir RE, et al. Rett syndrome is caused by mutations
in X-linked MECP2, encoding methyl-CpG-binding protein 2. Nat
Genet. 1999;23:185-188. 5 Tarquinio. Age of Diagnosis in Rett
Syndrome: Patterns of Recognition Among Diagnosticians and Risk
Factors for Late Diagnosis. Pediatric Neurology.
2015;52:585-591.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220912005299/en/
Media: Acadia Pharmaceuticals Inc. Deb Kazenelson (818) 395-3043
media@acadia-pharm.com
Investors: Acadia Pharmaceuticals Inc. Mark Johnson, CFA (858)
261-2771 ir@acadia-pharm.com
Acadia Pharmaceuticals (NASDAQ:ACAD)
Historical Stock Chart
From Mar 2024 to Apr 2024
Acadia Pharmaceuticals (NASDAQ:ACAD)
Historical Stock Chart
From Apr 2023 to Apr 2024