The results of the APPEAR-C3G study demonstrate the potential of Fabhalta in treating C3 glomerulopathy (C3G), an ultra-rare kidney disease with no approved treatments. Fabhalta showed sustained proteinuria reduction at 12 months, along with improvements in estimated glomerular filtration rate (eGFR) slope compared to patients’ historic rapid decline. The study also highlighted a favorable safety profile for Fabhalta, with no new safety signals reported.
These findings are significant for the management of C3G, as Fabhalta is the only oral alternative complement pathway inhibitor targeting the underlying cause of the disease. Approximately 50% of C3G patients progress to kidney failure within 10 years of diagnosis, emphasizing the need for effective treatments in this patient population.
Novartis is advancing its renal portfolio with regulatory submissions for Fabhalta completed in the EU, China, and Japan, and expected in the US by year-end. The company is committed to transforming patient care in kidney disease and continues to explore innovative treatment options for rare kidney diseases like C3G, IgA nephropathy, atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis, and lupus nephritis.
In addition to Fabhalta, Novartis is developing two other therapies for IgA nephropathy with unique mechanisms of action: atrasentan and zigakibart. These advancements in the renal portfolio aim to revolutionize the treatment paradigm for patients with rare kidney diseases and improve outcomes in this underserved patient population. Since then, we have continued to invest in research and development to address unmet needs in kidney diseases, including IgAN and C3G. Our commitment to patients with kidney diseases drives us to develop innovative treatments like FABHALTA to help improve outcomes and quality of life. We will continue to work tirelessly to advance the field of nephrology and make a meaningful difference in the lives of patients around the world.” Novartis is committed to advancing research and development in the field of renal diseases to preserve kidney function and improve the lives of those affected by such conditions. Our goal is to help individuals with kidney diseases live longer, without the need for dialysis or transplantation.
Please note that this press release contains forward-looking statements, which are subject to various risks and uncertainties. While we strive to improve and extend the lives of patients through our innovative medicines, there are factors that may impact the commercial success of our products. We encourage you to review our current Form 20-F for more information on these risks and uncertainties.
Novartis is dedicated to reimagining medicine and empowering patients, healthcare professionals, and societies in the face of serious diseases. With our global reach, we aim to make a positive impact on over 250 million people worldwide.
For more information on Novartis and our initiatives, visit us at https://www.novartis.com and https://www.novartis.us, and connect with us on LinkedIn, Facebook, Twitter, and Instagram.
References:
– Smith RJ, Kavanagh D, Vivarelli M, et al. Efficacy and safety of iptacopan in patients with C3 glomerulopathy: 12-month results from the Phase 3 APPEAR-C3G study.
– FABHALTA prescribing information. East Hanover, NJ: Novartis Pharmaceuticals Corp; August 2024.
– Schena FP, Esposito P, Rossini M. A Narrative Review on C3 Glomerulopathy: A Rare Renal Disease.
– Martín B, Smith RJH. In: Adam MP, Feldman J, Mirzaa GM, et al., editors. C3 Glomerulopathy. GeneReviews ® [Internet]. Updated 2018.
– Kidney Disease: Improving Global Outcomes (KDIGO) Glomerular Diseases Work Group. KDIGO 2021 Clinical Practice Guideline for the Management of Glomerular Diseases.
– Kavanagh D, Bomback A, Vivarelli M, et al. Efficacy and Safety of Iptacopan in Patients with C3 Glomerulopathy: Results from the Phase 3 APPEAR-C3G Trial.
– Smith RJH, Appel GB, Blom AM, et al. C3 Glomerulopathy – understanding a rare complement-driven renal disease.
– ClinicalTrials.gov. Study of Efficacy and Safety of Iptacopan in Patients With C3 Glomerulopathy. (APPEAR-C3G).
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– Sloan Simpson: +1 862 345 4440
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