For people living with IgA nephropathy (IgAN) – a kidney disease in which the immune system attacks the kidneys – the waiting game can stretch years. Despite the best current treatments, up to 50% of IgAN patients with significant protein in their urine will progress to kidney failure within 10 to 20 years, requiring dialysis or transplant. In August 2024, that landscape changed. The FDA approved a first-in-class medicine called Fabhalta (iptacopan), offering a new way to fight this disease.
What is IgA nephropathy and why is this approval important?
IgA nephropathy is a rare but progressive kidney disease. It’s caused when the immune system produces too much of a protein called IgA, which settles in the kidneys and triggers inflammation. Unlike lupus (which affects multiple organs), IgAN attacks the kidneys specifically. About 25 people per million worldwide are newly diagnosed each year, and every patient’s disease follows a unique path.
You can read the full original announcement from Novartis here: FDA approval for Fabhalta.
For decades, the main treatments for IgAN focused on managing symptoms – reducing blood pressure, controlling inflammation – but they didn’t address the root cause. Fabhalta works differently. It targets the “alternative complement pathway,” a part of the immune system that, when overly activated in IgAN, drives kidney damage. By inhibiting this pathway, Fabhalta reduces the amount of protein leaking into the urine – a marker of kidney damage.
What does the evidence show?
In the Phase III APPLAUSE-IgAN trial, patients taking Fabhalta showed a 44% reduction in proteinuria (protein in urine) at 9 months compared to baseline. The placebo group showed only a 9% reduction. That’s a clinically meaningful difference of 38% in favour of Fabhalta, with very strong statistical significance (p less than 0.0001).
The treatment also worked consistently across different patient groups – regardless of age, sex, race, or baseline kidney function – suggesting it has broad benefit.
New treatments with different mechanisms of action give doctors and patients more choices. Every person’s IgAN is unique, and targeted therapy means we can now select the right treatment for the right patient at the right time.
– Guy Hill, Chair of MRIKPA
Why this matters for kidney patients
For IgAN patients in the UK, Fabhalta represents a significant step forward. Reducing protein in the urine is important because proteinuria is both a sign and a driver of kidney damage. Less protein leaking into the urine means less inflammation and potentially slower progression toward kidney failure.
At MRIKPA, we know that kidney patients often feel powerless watching their kidney function decline month after month. A new medicine that slows that decline – or even stops it – offers real hope.
What this does – and does not – mean right now
Fabhalta was approved based on proteinuria reduction, but the critical question remains: does it slow kidney function decline long-term? The ongoing Phase III trial is measuring that now, with results expected in 2025. If those results confirm that Fabhalta slows kidney disease progression, the approval will become permanent. Until then, it’s approved on an accelerated basis – meaning it can be prescribed, but final approval depends on additional data.
Also important: Fabhalta does suppress part of the immune system, and patients must be vaccinated against certain bacterial infections before starting treatment. This is a real consideration that requires discussion with your renal team.
This is not a cure, and it may not work for every IgAN patient. But for those living with progressive IgAN, it offers a chance to slow kidney damage and potentially avoid or delay dialysis and transplant.
You can read the full original article from Novartis here: Novartis receives FDA accelerated approval for Fabhalta.
If you have IgAN and are interested in learning more about Fabhalta or other treatment options, speak with your kidney specialist. For general information about kidney disease management, visit our renal care page or explore kidney research and treatment advances.
Questions about IgAN or new treatment options? Get in touch at support@mrikpa.org.uk or call 07745 242 684. We’re here to help you understand your options.
Source: Novartis, August 2024 – read the original article
This article is provided for general information and awareness purposes only and was believed to be accurate at the time of publishing. It is not intended as medical advice – please always consult your doctor or renal team for guidance on your individual circumstances. Images used are for illustration purposes only and may not be medically or editorially accurate. While we take every care, errors can occur. If you spot an inaccuracy, please let us know at support@mrikpa.org.uk.
Source: Novartis, August 2024 – read the original announcement
