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EMA grants orphan drug designation to Novartis iptacopan in IgA nephropathy

Saturday, October 24, 2020, 09:00 Hrs  [IST]

Novartis announced that the European Medicines Agency (EMA) has granted an orphan drug designation for iptacopan (LNP023) in IgA nephropathy (IgAN), following a recommendation from the Committee for Orphan Medicinal Products (COMP).

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.

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 years. 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.

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.

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.

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 US FDA and EMA in C3G and PNH10 as well as EMA PRIME designation for C3G.


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