IgA Nephropathy ( IgAN)Īlso known as Berger’s disease, IgA nephropathy (IgAN) is a chronic kidney disease that damages glomeruli inside the kidneys. Moving forward, Novartis will continue to evaluate iptacopan in the Phase 3 APPLAUSE clinical trial. In addition to reducing proteinuria (excess protein in the urine), iptacopan improved kidney function.
Altogether, iptacopan was found to be relatively safe and well-tolerated. Iptacopan reached its primary endpoint within this clinical trial. Prior to the trial, researchers predicted that the highest iptacopan dose would result in a 23% proteinuria reduction. Ultimately, the primary endpoint was a significant proteinuria reduction at 90 days. Researchers wanted to determine the safety, efficacy, and tolerability of iptacopan. Patients received either varying iptacopan doses (up to 200mg 2x daily) or a placebo. Breakthrough Therapy designation. Iptacopan was granted Breakthrough Therapy designation from the FDA for the treatment of PNH.ĭuring the Phase 2 clinical trial, researchers evaluated iptacopan for patients with IgAN. In terms of PRIME designation, iptacopan received this from the EMA for C3G. Additionally, the therapy received Orphan Drug designations from the EMA and FDA for C3 glomerulonephropathy (C3G) and paroxysmal nocturnal hemoglobinuria. Iptacopan received Orphan Drug designation from the European Medicines Agency (EMA) in regards to IgAN. Beyond IgAN, researchers are also exploring iptacopan as a potential treatment for paroxysmal nocturnal hemoglobinuria (PNH) and membranous nephropathy (MN), among others. For example, up to 30% of patients with IgAN will have kidney failure within 10 years of diagnosis.īy acting as an immunomodulatory agent, iptacopan calms down complement overactivation and inhibits IgAN progression. Learning to treat complement-driven disorders is also important as these conditions affect young adults significantly more than other groups, causing an increased need for dialysis or kidney transplants. In the case of IgAN, this inflammation affects the kidneys. Thus, overactivation leads to inflammation and, ultimately, organ damage. Complement-driven disorders are those caused by overactivation of the alternative complement pathway. 
Upon administration, iptacopan binds to FB and prevents the formation of the alternative pathway (AP) C3-convertase (C3bBb), limits the cleavage of C3 to the active fragment C3b and may prevent C3b-mediated extravascular hemolysis in certain complement-driven disorders. The National Cancer Institute ( NCI) describes iptacopan as:Īn orally available, small-molecule inhibitor of complement factor B (FB) with potential immunomodulatory activity. As IgAN currently has no FDA-approved treatments, this data shows promise for the future of IgAN treatment. During the presentation, Novartis shared Phase 2 primary endpoint data regarding iptacopan (LNP023), an investigational therapy for patients with IgA nephropathy (IgAN). According to a press release, global medicines company Novartis presented data at the Congress. Though the event was held virtually, the meeting still expanded an understanding on research and advances within this field. From June 5-8, 2021, the European Renal Association – European Dialysis and Transplant Association (ERA-EDTA) held its 58th Annual Congress.
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