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MarketScreener Homepage  >  Equities  >  Swiss Exchange  >  Novartis AG    NOVN   CH0012005267

NOVARTIS AG

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Novartis : receives US Food and Drug Administration Orphan Drug Designation for branaplam in Huntington's disease

10/22/2020 | 09:18am EST

Basel - Novartis today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for branaplam (LMI070) in Huntington's disease (HD).

An Orphan Drug Designation grants special status to a drug that treats a rare disease or condition, and provides companies certain benefits to encourage the continued development of medicines that bring novel solutions to patients with these severe diseases.

In preclinical models, branaplam has been shown to reduce levels of mutant huntingtin protein. In addition, during the investigation of branaplam in spinal muscular atrophy (SMA), it was also observed to reduce huntingtin messenger RNA (mRNA) in SMA patients. A decrease of huntingtin mRNA is expected to result in reduction of huntingtin protein levels, the underlying cause of HD. Based on these findings, Novartis intends to start a development program for branaplam to determine if it has the potential to be a transformative treatment for people living with this devastating condition.

Current treatment options for HD are limited to symptomatic treatments and there are no approved disease modifying therapies that delay disease onset or slow progression of the disease.

Branaplam is currently under investigation for the treatment of spinal muscular atrophy (SMA). SMA is a rare, progressive genetic disease, characterized by loss of motor neurons that are responsible for muscle function. Branaplam is dosed once weekly for the treatment of SMA, and the same dosing regimen may also be a possibility for HD.

Novartis plans to start the Phase IIb trial for branaplam in HD patients in 2021.

About branaplam

Branaplam (LMI070) is a once-weekly, orally administered, small molecule RNA splicing modulator that is currently under investigation for the treatment of spinal muscular atrophy (SMA). SMA is a rare, progressive genetic disease, characterized by loss of motor neurons that are responsible for muscle function. Novartis has more than 5 years' clinical experience with branaplam in our ongoing SMA development program.

About Huntington's disease

Huntington's disease (HD) is a rare, inherited neurodegenerative disease that leads to progressive disability and death. Everyone has the huntingtin (HTT) gene, but only those who have a mutated form of the gene will develop the disease.

HD is characterized by progressive worsening in motor, cognitive and psychiatric symptoms. These symptoms usually appear between the ages of 30 to 50, and worsen over a 15-20-year period. Approximately 70,000 people across Europe and the US have been clinically diagnosed with HD.

Current treatment options for HD are limited to symptomatic treatments and there are no approved disease modifying therapies that delay disease onset or slow progression of the disease.

Disclaimer

This media update 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,' 'may,' 'could,' 'would,' 'expect,' 'anticipate,' 'seek,' '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 media update, 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 media update 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 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 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, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; 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 media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update as a result of new information, future events or otherwise.

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 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world.

Contact:

Antonio Ligi

Tel: +41 61 324 1374

Email: antonio.ligi@novartis.com

(C) 2020 Electronic News Publishing, source ENP Newswire

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Financials (USD)
Sales 2020 49 432 M - -
Net income 2020 8 326 M - -
Net Debt 2020 19 270 M - -
P/E ratio 2020 24,6x
Yield 2020 3,50%
Capitalization 206 B 205 B -
EV / Sales 2020 4,55x
EV / Sales 2021 4,20x
Nbr of Employees 110 000
Free-Float 86,9%
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Average target price 105,79 $
Last Close Price 90,44 $
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Spread / Lowest Target -28,4%
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Vasant Narasimhan Chief Executive Officer
Hans Jörg Reinhardt Independent Non-Executive Chairman
Harry Werner Kirsch Chief Financial Officer
John Tsai Chief Medical Officer
Elizabeth Theophille Chief Technology & Digital Officer
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