AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1
March 27 2018 - 8:05AM
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company
developing treatments for patients suffering from rare and
life-threatening neurological genetic diseases, today announced
that Japan’s Ministry of Health, Labour and Welfare (MHLW) awarded
the company’s initial product candidate, AVXS-101, SAKIGAKE
Designation (SAKIGAKE) for the treatment of spinal muscular atrophy
(SMA) Type 1. The designation was based on data from the Phase 1
clinical trial of the company’s proprietary gene therapy.
SAKIGAKE is intended to promote research and development in
Japan for innovative new medical products that satisfy certain
criteria, such as the severity of the intended indication, by
providing prioritized consultation review during the early stages
of development and by shortening the target review period for
license applications from 12 months to as few as six months. The
benefits of SAKIGAKE Designation are similar to the Breakthrough
Therapy Designation in the United States and access into the
PRIority MEdicines (PRIME) scheme in the EU, both of which have
already been granted to AVXS-101.
“This designation by the MHLW underscores the agreement by the
Japanese government that there is an urgent need for a new
therapeutic approach to treat Japanese patients diagnosed with SMA
Type 1, and allows for enhanced discussions between AveXis and the
agency to potentially expedite the timeline for approval of
AVXS-101,” said Sean Nolan, President and Chief Executive Officer
of AveXis.
James L’Italien, Chief Regulatory and Quality Officer of AveXis,
stated, “We look forward to continuing to work closely with the
Japanese authority as well as regulatory authorities around the
globe in our effort to bring this gene therapy to SMA patients and
their families as safely and efficiently as possible.”
About SAKIGAKE Designation The MHLW in
Japan formed the “Strategy of SAKIGAKE” in June 2014 in an effort
to facilitate the review and approval of innovative medical
products. The Strategy of SAKIGAKE consists of two
measurements – SAKIGAKE Designation and the Scheme for Rapid
Authorization of Unapproved Drugs – covering basic research to
clinical research and trials, approval reviews, safety measures,
insurance coverage, improvement of infrastructure and the
environment for corporate activities, and global expansion. The
SAKIGAKE Designation System intends to promote research and
development in Japan aiming at shortening premarket review period
for innovative new medical products that satisfy certain criteria,
such as severity of intended indication, by designating such
products during the early stages of development, and providing
prioritized consultation services and premarket pharmaceutical
affairs
review. For
more information, please visit Strategy of SAKIGAKE section of
http://www.mhlw.go.jp. About SMASMA is a severe
neuromuscular disease characterized by the loss of motor neurons
leading to progressive muscle weakness and paralysis. SMA is caused
by a genetic defect in the SMN1 gene that codes SMN, a protein
necessary for survival of motor neurons. The incidence of SMA is
approximately one in 10,000 live births and is the leading genetic
cause of infant mortality.
The most severe form of SMA is Type 1, a lethal genetic disorder
characterized by motor neuron loss and associated muscle
deterioration, which results in mortality or the need for permanent
ventilation support before the age of two for greater than 90
percent of patients. SMA Type 2 typically presents between six and
18 months of age, and those affected will never walk without
support and most will never stand without support. SMA Type 2
results in mortality in more than 30 percent of patients by the age
of 25.
About AVXS-101AveXis’ initial product
candidate, AVXS-101, is its proprietary gene therapy currently in
development for the one-time treatment of SMA Types 1 and 2,
designed to address the monogenic root cause of SMA and prevent
further muscle degeneration by addressing the defective and/or loss
of the primary SMN gene. AVXS-101 also targets motor neurons,
providing rapid onset of effect and crossing the blood brain
barrier to allow effective targeting of both central and systemic
features.
About AveXis, Inc.AveXis, Inc. is a
clinical-stage gene therapy company, dedicated to developing and
commercializing novel treatments for patients suffering from rare
and life-threatening neurological genetic diseases. Our initial
product candidate, AVXS-101, is our proprietary gene therapy
currently in development for the treatment of SMA Type 1, the
leading genetic cause of infant mortality, and SMA Type 2. The U.S.
Food and Drug Administration has granted AVXS-101 Orphan Drug
Designation for the treatment of all types of SMA and Breakthrough
Therapy Designation, as well as Fast Track Designation for the
treatment of SMA Type 1. In addition to developing AVXS-101 to
treat SMA Type 1 and Type 2, we also plan to develop other novel
treatments for rare neurological diseases, including Rett syndrome
and a genetic form of amyotrophic lateral sclerosis caused by
mutations in the superoxide dismutase 1 (SOD1) gene.
For additional information, please visit www.avexis.com.
Forward-Looking Statements This press release
contains "forward-looking statements," within the meaning of the
Private Securities Litigation Reform Act of 1995, regarding, among
other things, AveXis’ research, development and regulatory plans
for AVXS-101. Such forward-looking statements are based on current
expectations and involve inherent risks and uncertainties,
including factors that could delay, divert or change any of them,
and could cause actual results to differ materially from those
projected in its forward-looking statements. Meaningful factors
which could cause actual results to differ include, but are not
limited to, the scope, progress, expansion, and costs of developing
and commercializing AveXis’ product candidates and regulatory
developments in the U.S., EU and Japan, as well as other factors
discussed in the "Risk Factors" and the "Management's Discussion
and Analysis of Financial Condition and Results of Operations"
sections of AveXis’ Annual Report on Form 10-K for the year ended
December 31, 2017, filed with the SEC on February 28, 2018. In
addition to the risks described above and in the Annual Reports on
Form 10-K, Quarterly Reports on Form 10-Q, Current Reports on Form
8-K and other filings with the SEC, other unknown or unpredictable
factors also could affect AveXis’ results. There can be no
assurance that the actual results or developments anticipated by
AveXis will be realized or, even if substantially realized, that
they will have the expected consequences to, or effects on, AveXis.
Therefore, no assurance can be given that the outcomes stated in
such forward-looking statements and estimates will be achieved.
Media Inquiries:Lauren BarbieroW2O
Group646-564-2156lbarbiero@w2ogroup.com Investor Inquiries:Jim
GoffAveXis, Inc.650-862-4134jgoff@avexis.com
AVEXIS, INC. (NASDAQ:AVXS)
Historical Stock Chart
From Jul 2024 to Aug 2024
AVEXIS, INC. (NASDAQ:AVXS)
Historical Stock Chart
From Aug 2023 to Aug 2024