07.08.2019 00:08

Press Release: Novartis stands behind Zolgensma(R) (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years of age with spinal muscula...

Basel, August 6, 2019 -- Today the FDA released a statement addressing

data integrity issues with the Biologics License Application (BLA) for

Zolgensma(R) (onasemnogene abeparvovec-xioi). First and foremost, we are

fully confident in the safety, quality and efficacy of Zolgensma. The

FDA supports the continued marketing and use of Zolgensma for patients

with spinal muscular atrophy (SMA) less than 2 years of age. We maintain

that the totality of the evidence demonstrating the product's

effectiveness and its safety profile continue to provide compelling

evidence supporting an overall favorable benefit-risk profile. We remain

steadfast that this important treatment remain available to pediatric

patients with SMA less than 2 years of age.

On June 28(th), AveXis voluntarily self-disclosed to the FDA and

subsequently to other health authorities that some data previously

submitted to the agency as part of our BLA package was inaccurate.

Avexis had become aware of allegations of data manipulation in a

specific animal testing procedure used in the development of the

product. The assays in question were used for initial product testing

and are not currently used for commercial product release. An

investigation was immediately initiated to rapidly understand any

implications and address the situation. Once we had interim conclusions

from our investigations, we shared our findings with the FDA. As noted

by the FDA, the data in question were a small portion of our overall

submission and are limited to an older process no longer in use.

At no time during the investigation did the findings indicate issues

with product safety, efficacy or quality. We remain fully capable of

releasing high-quality, fully compliant Zolgensma to patients in need.

We have and will continue to work in close cooperation with the FDA to

appropriately update our submission and address any quality gaps

identified. We are committed to ensuring the highest levels of

transparency and integrity with health agencies, as well as with the

patients and providers we serve. We do not expect this to impact the

timing of our ongoing Zolgensma regulatory filings and development

programs. AveXis is committed to taking appropriate action to prevent

future incidents across its portfolio of development programs.

About Zolgensma(R) (onasemnogene abeparvovec-xioi)

Zolgensma (onasemnogene abeparvovec-xioi) is a proprietary gene therapy

approved by the US Food and Drug Administration for the treatment of

pediatric patients less than 2years of age with spinal muscular atrophy

(SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1)

gene. Zolgensma is designed to address the genetic root cause of SMA by

providing a functional copy of the human SMN gene to halt disease

progression through sustained SMN protein expression with a single,

one-time intravenous (IV) infusion. Zolgensma represents the first

approved therapeutic in a proprietary platform to treat rare, monogenic

diseases using gene therapy. The therapy is also anticipated to receive

approval in Japan and the European Union later this year.

About Spinal Muscular Atrophy (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.(8,9) The incidence of SMA is

approximately 1 in 10,000 live births and it is the leading genetic

cause of infant mortality.(9,10) The most severe form of SMA is Type 1,

a lethal genetic disorder characterized by rapid motor neuron loss and

associated muscle deterioration, resulting in mortality or the need for

permanent ventilation support by 24 months of age for more than 90

percent of patients if left untreated.(11)


Zolgensma (onasemnogene abeparvovec-xioi) is an adeno-associated virus

vector-based gene therapy indicated for the treatment of pediatric

patient less than 2 years of age with spinal muscular atrophy (SMA) with

bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

Limitation of Use:

The safety and effectiveness of repeat administration of Zolgensma have

not been evaluated.

The use of Zolgensma in patients with advanced SMA (e.g., complete

paralysis of limbs, permanent ventilator-dependence) has not been


Important Safety Information

Acute Serious Liver Injury

Acute serious liver injury and elevated aminotransferases can occur with

Zolgensma. Patients with pre-existing liver impairment may be at higher

risk. Prior to infusion, assess liver function of all patients by

clinical examination and laboratory testing (e.g., hepatic

aminotransferases [aspartate aminotransferase and alanine

aminotransferase], total bilirubin and prothrombin time). Administer

systemic corticosteroid to all patients before and after Zolgensma

infusion. Continue to monitor liver function for at least 3 months after



Transient decreases in platelet counts, some of which met the criteria

for thrombocytopenia, were observed at different time points after

Zolgensma infusion. Monitor platelet counts before Zolgensma infusion

and on a regular basis afterwards.

Elevated Troponin-I

Transient increases in cardiac troponin-I levels (up to 0.176 mcg/L)

were observed following Zolgensma infusion in clinical trials. The

clinical importance of these findings is not known. However, cardiac

toxicity was observed in animal studies. Monitor troponin-I before

Zolgensma infusion and on a regular basis for at least 3 months


Adverse Reactions

The most commonly observed adverse reactions (incidence >=5%) were

elevated aminotransferases and vomiting.

Please read full Prescribing Information: for

Zolgensma, including Boxed Warning for Acute Serious Liver Injury.


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 "stands behind," "confident," "supports," "supporting," "remain

steadfast," "will," "committed," "expect," "ongoing," "anticipated,"

"later this year," "potential," "expectations," or similar terms, or by

express or implied discussions regarding potential marketing approvals,

new indications or labeling for Zolgensma, or regarding potential future

revenues from Zolgensma. 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 Zolgensma will be submitted or approved for sale or for

any additional indications or labeling in any market, or at any

particular time. Nor can there be any guarantee that Zolgensma will be

commercially successful in the future. In particular, our expectations

regarding Zolgensma 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 and requirements

for increased pricing transparency; 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, data integrity 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


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 more than 750 million people

globally and we are finding innovative ways to expand access to our

latest treatments. About 108,000 people of more than 140 nationalities

work at Novartis around the world. Find out more at

Novartis is on Twitter. Sign up to follow @Novartis at or follow @NovartisNews for the latest News

& Media Updates at

For Novartis multimedia content, please visit

For questions about the site or required registration, please contact

# # #

Novartis Global External Communications


Eric Althoff Farah Bulsara Speer

Novartis US External Communications VP, Corporate Communications, AveXis

+1 646 438 4335 +1 312 543 2881 (mobile)

Sreejit Mohan

Novartis Global External Communications

+41 79 747 9157

(MORE TO FOLLOW) Dow Jones Newswires

August 06, 2019 18:08 ET (22:08 GMT)

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