NOVARTIS AG The breakthrough designation is intended to expedite the development and review of medicines for serious conditions to address unmet medical need, where early clinical evidence indicates a drug may demonstrate substantial improvement over available therapy on clinically significant endpoints. The FDA granted BTD to iptacopan for the treatment of PNH based on positive interim results from two ongoing Phase II studies, where iptacopan showed substantial benefits both in patients who remained anemic and dependent on transfusions despite standard of care anti-complement treatment8, as well as monotherapy in anti-C5 naive PNH patients.
PNH is a rare and life threatening blood disorder characterized by complement-driven hemolysis, thrombosis and impaired bone marrow function9,10, resulting in anemia, fatigue and other debilitating symptoms that can impact patients' quality of life1-3. Despite current standard of care - anti-C5 therapy eculizumab or ravulizumab - a large proportion of PNH patients remain anemic and dependent on transfusions1,2,9,11,12.
FDA grants the rare pediatric designation for serious or life-threatening diseases primarily affecting individuals aged 18 years or younger and impacting fewer than 200,000 people. C3G, an ultra-rare and severe form of primary glomerulonephritis4,13, is characterized by complement dysregulation. It has a poor prognosis; about 50% of patients progress to end-stage renal disease (ESRD) within 10 years, and 50-70% experience disease recurrence post kidney transplant14. It has a worldwide annual incidence of 1-2 per million15.
About iptacopan
Iptacopan (LNP023) is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway6,7. It is currently in clinical development for PNH, as well as C3G and a number of other renal conditions with complement system involvement where significant unmet needs exist, including IgA nephropathy (IgAN), atypical hemolytic uremic syndrome (aHUS), and membranous nephropathy (MN).
Positive Phase II data from one study in PNH were presented at the
Iptacopan has the potential to become the first complement pathway inhibitor to slow disease progression in a number of complement-driven diseases. Based on disease prevalence and the positive interim data from Phase II studies, iptacopan has also received orphan drug designations from the FDA and EMA in C3G and PNH16, as well as
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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 110,000 people of more than 140 nationalities work at Novartis around the world.
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