Have you heard the latest on treatment options for rare kidney diseases? Novartis’ Fabhalta (iptacopan) has received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). This is for the Fabhalta C3 glomerulopathy treatment in adults with C3 glomerulopathy (C3G), a rare and progressive kidney disease. Meaning, this will make Fabhalta the first medicine specifically targeting the underlying cause of C3G. The Phase III APPEAR-C3G trial demonstrated a significant reduction in proteinuria and stabilization of kidney function in patients treated with Fabhalta.

Doctors mostly diagnose young adults with C3 glomerulopathy, a rare kidney disease. About half of patients progress to kidney failure within a decade of diagnosis. Currently, there are no approved treatments for C3G, making Fabhalta’s potential approval a significant milestone. The European Medicines Agency (EMA) and FDA have participated in the regulatory process for Fabhalta in various indications. This highlights its potential impact on rare diseases.

What you need to know

The new indications for Fabhalta are:

– Paroxysmal nocturnal haemoglobinuria. Novartis’ Fabhalta is indicated as monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

– Complement 3 glomerulopathy. Fabhalta is indicated for the treatment of adult patients with complement 3 glomerulopathy (C3G) in combination with a renin-angiotensin system (RAS) inhibitor, or in patients who are RAS-inhibitor intolerant, or for whom a RAS inhibitor is contraindicated.

Novartis’ Fabhalta will become the first approved treatment for C3G. It addresses a significant unmet need for patients with this condition. The APPEAR-C3G trial showed a 35.1% reduction in proteinuria, a key marker for kidney disease progression. It also demonstrated stabilization of kidney function. Novartis is also exploring Fabhalta for multiple complement-mediated diseases. These include IgA nephropathy, atypical hemolytic uremic syndrome, and others. For more information on this groundbreaking treatment, you can refer to the detailed report here.

The potential approval of Fabhalta for C3G may have implications for health economics and outcomes research. By reducing proteinuria and stabilizing kidney function, Fabhalta may delay or prevent kidney failure. This could reduce the need for dialysis and transplantation. The introduction of a targeted therapy could lead to cost savings.

Novartis’ focus on complement-mediated diseases highlights the importance of targeted therapies. These therapies address unmet needs in rare diseases, potentially influencing future research directions and investment in similar areas.