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Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1

Novartis International AG /
Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a
one-time treatment designed to address the genetic root cause of SMA Type 1
. Processed and transmitted by West Corporation.
The issuer is solely responsible for the content of this announcement.

* The AVXS-101, now known as ZOLGENSMA(®) (onasemnogene abeparvovec-xxxx)[1],
filing is supported by data from the START trial which demonstrated a
dramatic increase in survival and transformative improvement in achievement
of developmental milestones compared to the natural history of SMA Type 1[2]

* SMA Type 1 is a progressive neuromuscular disease and the leading cause of
genetic mortality in infants globally [3]

* ZOLGENSMA(®) represents the first in a proprietary platform to treat rare,
monogenic diseases using gene replacement therapy - technology that replaces
a missing or defective gene with a functional copy to correct the underlying
cause of genetic disease

Basel, December 3, 2018 - Novartis today announced that the U.S. Food and Drug
Administration (FDA) has accepted the company's Biologics License Application
(BLA) for AVXS-101, now known as ZOLGENSMA(®) (onasemnogene abeparvovec-
xxxx)[1], an investigational gene replacement therapy for the treatment of
spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the
genetic root cause of SMA Type 1, a deadly neuromuscular disease with limited
treatment options. ZOLGENSMA previously received Breakthrough Therapy
designation and has been granted Priority Review by the FDA, with regulatory
action anticipated in May 2019.

SMA is caused by a defective or missing SMN1 gene.[4] Without a functional SMN1
gene, infants with SMA Type 1 rapidly lose the motor neurons responsible for
muscle functions such as breathing, swallowing, speaking and walking.[3] Left
untreated, a baby's muscles become progressively weaker eventually leading to
paralysis or death, in most cases by his or her second birthday.[5] Delivered as
a single, one-time infusion, this breakthrough technology works by replacing the
missing or defective SMN1 gene with a functional copy that makes SMN protein,
thereby improving motor neuron function and survival.[2]

"This important step by the FDA brings us ever closer to delivering ZOLGENSMA to
patients with SMA Type 1. Babies affected by this rare disease are currently
faced with debilitating disease progression and lifelong invasive chronic
treatment. As a one-time infusion that addresses the genetic root cause of SMA
without the need for repeat dosing, ZOLGENSMA represents a potentially
significant therapeutic advance for these patients and their families," said
David Lennon, president of AveXis. "The introduction of one-time, potentially
curative therapies will require rethinking how our healthcare system manages
diagnosis, treatment, care and associated costs for patients with genetic
disease. Novartis and AveXis are proud to lead the way toward a modern
healthcare system built on the tremendous value of truly innovative and
transformative medicines that could bend the curve of life. We are committed to
flexibly partnering with healthcare stakeholders to ensure appropriate access to
our medicines."

In the START trial, all 15 patients infused with ZOLGENSMA were alive and
without the need for permanent ventilation* at 24 months. Ninety-two percent
(11/12) of patients who received the proposed therapeutic dose of ZOLGENSMA
could sit unassisted for >=5 seconds, a milestone never achieved in the natural
history of SMA Type 1. Natural history indicates that more than 90 percent of
untreated patients with SMA Type 1 will die or require permanent ventilation by
24 months of age.[5] Patients who voluntarily enrolled in an ongoing
observational long-term follow-up of the START trial have maintained their
developmental motor milestones - including patients who are four years post
infusion - with some achieving additional motor milestones. The most commonly
observed side effect in the ZOLGENSMA clinical trial was elevated liver

In Japan, where ZOLGENSMA has SAKIGAKE Designation, a decision by regulators on
the New Drug Application (J-NDA) is expected in the first half of 2019. In
Europe, where ZOLGENSMA has PRIME (PRIority MEdicines) designation, a decision
by regulators on the Marketing Authorization Application (MAA) is expected in
mid-2019. The SAKIGAKE and PRIME designations are comparable to the FDA's
Breakthrough Therapy designation. These regulatory applications are based
primarily on data from the START trial.

Priority Review designation means the FDA's goal is to take action on an
application within six months, compared to 10 months under standard review.

About SMA
SMA 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.[3] The incidence of SMA is approximately one in 10,000 live
births and is the leading genetic cause of infant mortality. [7] 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 by 24 months of age for more than 90 percent
of patients. [3],[5]

ZOLGENSMA ([onasemnogene abeparvovec-xxxx[1];] AVXS-101) is a proprietary gene
replacement therapy currently in development as a one-time infusion for SMA Type
1. ZOLGENSMA is designed to address the monogenic root cause of SMA and prevent
further muscle degeneration by replacing the defective and/or loss of the
primary SMN gene (SMN1).

About the START Trial [2],[6]
START was a Phase 1 study evaluating safety and efficacy of ZOLGENSMA in SMA
Type 1 patients genetically tested to confirm bi-allelic SMN1 deletions, 2
copies of survival motor neuron 2 (SMN2), negative findings for the c.859G>C
modification in exon 7 and with the onset of clinical symptoms before 6 months
of age. ZOLGENSMA was delivered intravenously during a single-dose infusion in
patients 0.9 to 7.9 months of age. Two cohorts were dosed: Cohort 1 (n=3)
received the low dose used in this study and Cohort 2 (n=12) received the high
dose used in this study.

At the 24-month follow up, all 15 patients (100%), who were over all 24 months
of age, were event-free, as opposed to only 8% of patients in a natural history
study. This indicates a significant and clinically meaningful increase in
overall survival for patients infused with ZOLGENSMA when compared to untreated
patients. At two years following infusion, no patient deaths were reported. The
most commonly observed side effect in the ZOLGENSMA clinical trial was elevated
liver enzymes.

The reported study outcomes reflect Cohort 2 and includes follow-up of all
patients out to 24 months following ZOLGENSMA infusion. Patients in Cohort 2
consistently achieved and maintained key developmental motor milestones. At 24
months of follow-up post-infusion, 11 patients (91.7%) were able to hold their
head erect for >= 3 seconds and sit without support for >= 5 seconds, 10
patients (83.3%) were able to sit without support for >= 10 seconds, 9 patients
(75.0%) were able to sit without support for >= 30 seconds and 2 patients each
(16.7%) were able to stand alone, walk with assistance and walk alone.

Of the 10 patients in Cohort 2 that were not using non-invasive ventilation
(NIV) at baseline, 7 were free of daily NIV use at 24 months of follow-up.
Nearly all patients experienced common childhood respiratory illnesses that, in
children with SMA Type 1, typically result in tracheostomy or death. All
patients survived respiratory hospitalizations without tracheostomy or the need
for permanent ventilation.

Nutritional gains were also observed. In Cohort 2, seven patients did not
receive enteral feeding prior to ZOLGENSMA infusion. One of these 7 patients had
nutritional support post-ZOLGENSMA infusion to assist wound healing following a
difficult recovery from scoliosis surgery but was also feeding orally. Four of
the 5 patients in Cohort 2 who received enteral feeding prior to ZOLGENSMA
infusion were able to feed orally at end of study; thus, a total of 11 of the
12 patients in Cohort 2 were able to feed orally, 6 exclusively.

Patients receiving the therapeutic dose achieved statistically significant motor
function improvements by month 1 and month 3; Children's Hospital of
Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) mean increases
from baseline were 9.8 points (n=12, P < 0.001) and 15.4 points (n=12, P <
0.001), respectively. Motor function improvements were sustained over time in
patients infused with ZOLGENSMA. Eleven of twelve (91.7%) Cohort 2 patients
achieved a >= 50 CHOP-INTEND during the 24-month study period. Early
intervention and dose appear to positively affect the response. In general
clinical practice, untreated SMA Type 1 children 6 months of age or older do not
surpass a score of 40 points on the CHOP-INTEND. Furthermore, an average decline
of 10.7 points between the ages of 6 and 12 months were reported amongst
untreated infants followed as part of a prospective natural history.

Cohort 2 patients who are currently voluntarily enrolled in an ongoing
observational long-term follow-up of this study have maintained their
developmental motor milestones - including patients who are four years post
infusion - with some achieving additional motor milestones. Four patients
attained new milestones, including 2 patients who sat unassisted for >=30
seconds and two patients were able to stand with support.

This press release contains forward-looking statements within the meaning of the
United States Private Securities Litigation Reform Act of 1995. Forward-looking
statements can generally be identified by words such as "potential," "can,"
"will," "plan," "expect," "anticipate," "look forward," "believe," "committed,"
"investigational," "pipeline," "launch," or similar terms, or by express or
implied discussions regarding potential marketing approvals, new indications or
labeling for the investigational or approved products described in this press
release, or regarding potential future revenues from such products. You should
not place undue reliance on these statements. Such forward-looking statements
are based on our current beliefs and expectations regarding future events, and
are subject to significant known and unknown risks and uncertainties. Should one
or more of these risks or uncertainties materialize, or should underlying
assumptions prove incorrect, actual results may vary materially from those set
forth in the forward-looking statements. There can be no guarantee that the
investigational or approved products described in this press release will be
submitted or approved for sale or for any additional indications or labelling in
any market, or at any particular time. Nor can there be any guarantee that such
products will be commercially successful in the future. In particular, our
expectations regarding such products could be affected by, among other things,
the uncertainties inherent in research and development, including clinical trial
results and additional analysis of existing clinical data; regulatory actions or
delays or government regulation generally; global trends toward health care cost
containment, including government, payor and general public pricing and
reimbursement pressures; our ability to obtain or maintain proprietary
intellectual property protection; the particular prescribing preferences of
physicians and patients; general political and economic conditions; safety,
quality or manufacturing issues; potential or actual data security and data
privacy breaches, or disruptions of our information technology systems, and
other risks and factors referred to in Novartis AG's current Form 20-F on file
with the US Securities and Exchange Commission. Novartis is providing the
information in this press release as of this date and does not undertake any
obligation to update any forward-looking statements contained in this press
release as a result of new information, future events or otherwise.

*An event is defined as either death or at least 16 hours per day of required
ventilation support for breathing for 14 consecutive days in the absence of
acute reversible illness or perioperative change. [2]

About AveXis
AveXis, a Novartis company, is dedicated to developing and commercializing novel
treatments for patients suffering from rare and life-threatening neurological
genetic diseases. Our initial product candidate, ZOLGENSMA, is its proprietary
gene therapy currently in development for the treatment of spinal muscular
atrophy, or SMA. In addition to developing ZOLGENSMA to treat SMA, AveXis also
plans 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.

About Novartis
Novartis is reimagining medicine to improve and extend people's lives. As a
leading global medicines company, we use innovative science and digital
technologies to create transformative treatments in areas of great medical need.
In our quest to find new medicines, we consistently rank among the world's top
companies investing in research and development. Novartis products reach nearly
1 billion people globally and we are finding innovative ways to expand access to
our latest treatments. About 125 000 people of more than 140 nationalities work
at Novartis around the world. Find out more at www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at
For Novartis multimedia content, please visit www.novartis.com/news/media-
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   [1]   The brand name ZOLGENSMA® (onasemnogene abeparvovec-xxxx) has been
provisionally approved by the FDA for the investigational product AVXS-101 (with
a four-letter suffix to be added), but the product itself has not received
marketing authorization or BLA approval from any regulatory authorities.
   [2]   Mendell, JR., Al-Zaidy S., Shell R., et al. Single-dose gene-
replacement therapy for spinal muscular atrophy. N Engl J Med
2017; 377:1713-1722.
   [3]   Farrar MA, Park SB, Vucic S, et al. Emerging therapies and challenges
in spinal muscular atrophy. Ann Neurol. 2017; 81(3):355-368.
   [4]   Anderton RS and Mastaglia FL. Advances and challenges in developing a
therapy for spinal muscular atrophy. Expert Rev Neurother. 2015;15(8):895-908
   [5]   Finkel RS, McDermott MP, Kaufmann P. et al. Observational study of
spinal muscular atrophy type I and implications for clinical trials. Neurology.
   [6]   Mendell JR, Al Zaidy S, Shell R., et al. AVXS-101 Phase 1 Gene
Replacement Therapy Clinical Trial in SMA Type 1: Event-Free Survival and
Achievement of Developmental Milestones After 24 Months Post-Dosing. April 2018.
   [7]   National Organization for Rare Disorders (NORD). Spinal Muscular
Atrophy http://rarediseases.org/rare-diseases/spinal-muscular-atrophy/. Accessed
October 9, 2018.

# # #

Novartis Media Relations
Central media line: +41 61 324 2200
E-mail: media.relations@novartis.com

Eric Althoff Farah Speer
Novartis Global Media Relations AveXis
+41 61 324 7999 (direct) +1 312 543 2881(mobile)
+41 79 593 4202 (mobile) fspeer259@avexis.com

Novartis Investor Relations
Central investor relations line: +41 61 324 7944
E-mail: investor.relations@novartis.com

Central   North America

Samir Shah +41 61 324 7944 Richard Pulik +1 212 830 2448

Pierre-Michel Bringer +41 61 324 1065 Cory Twining +1 212 830 2417

Thomas Hungerbuehler +41 61 324 8425

Isabella Zinck +41 61 324 7188

Media release (PDF):

This announcement is distributed by West Corporation on behalf of West Corporation clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.

Source: Novartis International AG via GlobeNewswire

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