In the context of clinical trials, Novartis has achieved a significant milestone with its investigational oral factor B inhibitor during the phase 3 trial for kidney disorders. This triumph has spurred the pharmaceutical company into action as it prepares to initiate the process of requesting expedited FDA approval in the upcoming year.
The 9-month data stems from the APPLAUSE-IgAN study conducted by the Swiss pharmaceutical company. Comprehensive top-line results from this study are anticipated to be unveiled in the year 2025. This rigorous double-blind, placebo-controlled trial has been meticulously structured to assess the effectiveness and safety of administering oral iptacopan twice daily to a cohort of 470 adult participants who are grappling with IgA nephropathy (IgAN), a persistent kidney ailment sometimes referred to as Berger’s disease.
According to Novartis, the treatment achieved the study’s intermediate primary end aim, exhibiting statistically significant improvement in proteinuria reduction as compared to the placebo. However, the company did not offer any specific numbers. The projected slope of the glomerular filtration rate over the course of two years will be measured while research into the pill’s capacity to halt the advancement of IgAN proceeds.
The performance of iptacopan was described as “faultless” in a study that was published on October 2 by the financial analyst firm ODDO BHF. According to this company, the data represents a home run for Novartis.
According to Novartis, not only does iptacopan have a strong chance to become the first oral treatment for individuals diagnosed with IgAN, but it is also capable of going on to become the first specifically designed medication for IgAN that slows or prevents the development of kidney failure by engaging the complement system.
APPLAUSE-IgAN showed a safety profile that was similar to the findings that had been previously published from two phase 3 trials for paroxysmal nocturnal hemoglobinuria (PNH). Novartis has submitted iptacopan to authorities for the rare illness indication based on the results of those studies, and the company anticipates receiving a response in the year 2024.
Novartis will give AstraZeneca’s rare illness subsidiary, Alexion, a run for its money if the drug is authorized for use in PNH. It will compete against the complement inhibitors Soliris and Ultomiris. In a PNH experiment that took place some time ago, iptacopan showed superior performance versus Soliris.
ODDO BHF experts believe that there is “pipeline in a pill” potential with iptacopan. The tablet is also being worked on to treat a number of additional renal and hematological conditions for which there are very few treatments available – and even they are simply not good enough. When taking all signs into consideration, ODDO BHF forecasts that the initial launch of iptacopan will take place in 2024 and that peak sales will exceed $3 billion.