Anavex Life Sciences Receives Rare Pediatric Disease Designation from FDA for ANAVEX®2-73 (blarcamesine) for the Treatment o...
November 14 2019 - 07:00AM
Anavex Life Sciences Corp. (“Anavex” or the “Company”) (Nasdaq:
AVXL), a clinical-stage biopharmaceutical company developing
differentiated therapeutics for the treatment of neurodegenerative
and neurodevelopmental disorders including Alzheimer’s disease,
Parkinson’s disease, Rett syndrome and other central nervous system
(CNS) diseases, today announced that it received the Rare Pediatric
Disease (RPD) designation from the U.S. Food and Drug
Administration (FDA) for ANAVEX®2-73 (blarcamesine) for the
treatment of Rett syndrome, a rare genetic neurological disorder
associated with severe physical and cognitive impairments that
afflicts mostly girls.
The RPD designation provides priority review by
the FDA to encourage treatments for rare pediatric
diseases. Under the RPD program, a sponsor who receives an approval
for a drug for a "rare pediatric disease" may qualify for a voucher
that can be redeemed to receive a priority review by
the FDA of a subsequent marketing application for a
different product.
“We are pleased to have received the rare
pediatric designation from the U.S. FDA for ANAVEX®2-73
(blarcamesine) for young patients with Rett syndrome,” said
Christopher U. Missling, Ph.D., Chief Executive Officer of Anavex.
“This designation for ANAVEX®2-73 (blarcamesine) underscores the
significant unmet medical need for patients with this genetic
disease affecting nearly every aspect of the child’s life: their
ability to speak, walk, eat and easily breathe. Our goal is to
advance a potential treatment for Rett syndrome in order to bring
medicines to patients as soon as possible.”
About Rett Syndrome Rett
syndrome is a devastating, non-inherited genetic post-natal
progressive neurodevelopmental disorder that occurs almost
exclusively in girls and leads to severe impairments, affecting
nearly every aspect of the child’s life: their ability to speak,
walk, eat and easily breathe. The hallmark of Rett syndrome is near
constant repetitive hand movements while awake. The disease is
characterized by normal early growth and development (6 to 18
months) followed by a slowing of development, loss of purposeful
use of the hands, distinctive hand movements, autistic features,
slowed brain and head growth, ataxia, seizures and intellectual
disability.
Rett syndrome is caused by mutations in the
MECP2 gene and strikes all racial and ethnic groups. The disease
occurs worldwide in approximately one in every 10,000 to 15,000
live births. There is currently no cure for Rett syndrome.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a
publicly traded biopharmaceutical company dedicated to the
development of differentiated therapeutics for the treatment of
neurodegenerative and neurodevelopmental disorders including
Alzheimer’s disease, Parkinson’s disease, Rett syndrome and other
central nervous system (CNS) diseases, pain and various types of
cancer. Anavex’s lead drug candidate, ANAVEX®2-73 (blarcamesine),
recently completed a successful Phase 2a clinical trial for
Alzheimer’s disease. ANAVEX®2-73 (blarcamesine) is an orally
available drug candidate that restores cellular homeostasis by
targeting sigma-1 and muscarinic receptors. Preclinical studies
demonstrated its potential to halt and/or reverse the course of
Alzheimer’s disease. ANAVEX®2-73 (blarcamesine) also exhibited
anticonvulsant, anti-amnesic, neuroprotective and anti-depressant
properties in animal models, indicating its potential to treat
additional CNS disorders, including epilepsy. The Michael J. Fox
Foundation for Parkinson’s Research previously awarded Anavex a
research grant, which fully funded a preclinical study to develop
ANAVEX®2-73 (blarcamesine) for the treatment of Parkinson’s
disease. ANAVEX®3-71, which targets sigma-1 and muscarinic
receptors, is a promising preclinical drug candidate demonstrating
disease-modifying activity against the major hallmarks of
Alzheimer’s disease in transgenic (3xTg-AD) mice, including
cognitive deficits, amyloid and tau pathologies. In preclinical
trials, ANAVEX®3-71 has shown beneficial effects on mitochondrial
dysfunction and neuroinflammation. Further information is available
at www.anavex.com. You can also connect with the company
on Twitter, Facebook and LinkedIn.
Forward-Looking Statements
Statements in this press release that are not
strictly historical in nature are forward-looking statements. 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 set forth in the Company’s most recent Annual Report on Form
10-K filed with the SEC. Readers are cautioned not to place undue
reliance on these forward-looking statements, which speak only as
of the date hereof. All forward-looking statements are qualified in
their entirety by this cautionary statement and Anavex Life
Sciences Corp. undertakes no obligation to revise or update this
press release to reflect events or circumstances after the date
hereof.
For Further Information:
Anavex Life Sciences Corp.Research &
Business DevelopmentToll-free: 1-844-689-3939Email:
info@anavex.com
Investors & Media:Email:
ir@anavex.com
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