AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy T...
July 20 2016 - 7:00AM
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 the
U.S. Food and Drug Administration (FDA) has granted Breakthrough
Therapy Designation for AVXS-101, the company’s lead development
candidate for the treatment of spinal muscular atrophy (SMA) Type 1
in pediatric patients.
The Breakthrough Therapy Designation is based on preliminary
clinical results from the ongoing trial of AVXS-101, conducted in
collaboration with The Research Institute at Nationwide Children’s
Hospital and The Ohio State University.
“We are encouraged to have received Breakthrough Therapy
Designation for AVXS-101, and look forward to collaborating with
the FDA to determine next steps in the development pathway for
AVXS-101,” said Sean P. Nolan, president and chief executive
officer, AveXis. “By this action the FDA recognizes the high unmet
need for effective treatment options for patients suffering from
SMA.”
The FDA has requested the company submit a Type B meeting
request for a multidisciplinary, comprehensive discussion of the
development program for AVXS-101. The company plans to submit the
meeting request later this month.
SMA, which causes motor neuron loss and weakness, is the most
common genetic cause of infant death. The most severe form of SMA
is Type 1, which accounts for approximately 60 percent of cases at
birth, results in an inability to sit without support, difficulty
in breathing and swallowing. Furthermore, it results in death or
the need for permanent ventilation for greater than 90 percent of
patients by two years of age.
About Breakthrough Therapy Designation The
FDA’s Breakthrough Therapy Designation was established to
facilitate dialogue between FDA and the sponsor to provide advice
on generating evidence needed to support approval of the therapy in
an efficient manner – with more intensive and interactive guidance
on an efficient drug development program, an organizational
commitment involving FDA’s senior managers, and eligibility for
rolling review and priority review.
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.
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 1 is the leading genetic cause of
infant mortality.
About AVXS-101AVXS-101 is a proprietary gene
therapy candidate of a one-time treatment for SMA Type 1 and is the
only clinical-stage gene therapy in development for SMA. AVXS-101
is designed to address the monogenetic 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 crosses the blood
brain barrier allowing an IV dosing route and effective targeting
of both central and systemic features.
About AveXis, Inc.AveXis is a clinical-stage
gene therapy company developing treatments for patients suffering
from rare and life-threatening neurological genetic diseases. The
company’s initial proprietary gene therapy candidate, AVXS-101, is
in an ongoing Phase 1 clinical trial for the treatment of SMA Type
1. For additional information, please visit www.avexis.com.
Forward-Looking StatementsThis 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, including the expected timing for submitting a Type B
meeting request to the FDA and the potential for accelerated
development and review of 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 risk that Breakthrough Therapy Designation
could be withdrawn by the FDA in the future based on future
clinical results and regulatory developments in the United States
and foreign countries, as well as other factors discussed in the
"Risk Factors" and "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, 2015,
filed with the SEC on March 18, 2016. 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.
All forward-looking statements contained in this press release
are expressly qualified by the cautionary statements contained or
referred to herein. AveXis cautions investors not to rely too
heavily on the forward-looking statements AveXis makes or that are
made on its behalf. These forward-looking statements speak only as
of the date of this press release (unless another date is
indicated). AveXis undertakes no obligation, and specifically
declines any obligation, to publicly update or revise any such
forward-looking statements, whether as a result of new information,
future events or otherwise, except as required by law.
Media Inquiries:
Lauren Barbiero
W2O Group
646-564-2156
lbarbiero@w2ogroup.com
Investor Inquiries:
Jim Goff
AveXis, Inc.
650-862-4134
jgoff@avexis.com
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