NOVARTIS AG Orphan drug designation is granted to medicines that treat, prevent or diagnose a life-threatening or chronically debilitating rare disease, with a prevalence in the EU of below 5 in 10,000, and with either no currently approved method of diagnosis, prevention or treatment or with significant benefit to those affected by the disease.
IgAN is the most common form of primary glomerulonephritis an inflammatory kidney disease where abnormal IgA antibody is formed, which results in immune complex deposition in the glomerular mesangium, leading to deteriorating kidney function.1,2
In patients with IgAN, proteinuria is recognized as an independent risk factor of poor prognosis. Around 30% of patients with persistent proteinuria progress to end stage renal disease (ESRD) within 10 years5. No approved targeted therapy is available.
ESRD requires dialysis or kidney transplant and is associated with significant risk of complications, considerable impact on quality of life as well as an increased risk of premature death.5
About iptacopan
Iptacopan (LNP023) is a first-in-class oral, small-molecule, selective inhibitor of factor B, a key serine protease of the alternative pathway of the complement cascade mediating inflammatory responses.6-8
In addition to IgAN, iptacopan is in parallel development for a number of other renal conditions with complement system involvement where significant unmet needs exist, including C3 glomerulopathy (C3G), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (iMN). Novartis is also investigating iptacopan in paroxysmal nocturnal hemoglobinuria (PNH), a hematological disease.
Positive Phase II data in PNH were presented at the
Iptacopan has the potential to become the first alternative complement pathway inhibitor to slow disease progression in a number of complement-driven diseases. Based on disease prevalence and the interim data from Phase II studies, iptacopan has also received orphan drug designations from the FDA and EMA in C3G and PNH10 as well as
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
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:
Tel: +41 61 324 2279
Email: anja.vontreskow@novartis.com
(C) 2020 Electronic News Publishing, source
