HQ Team
August 8, 2024: Switzerland’s Novartis AG has received accelerated approval from the USFDA for its drug to reduce elevated levels of proteins in the urine of patients with a rare type of kidney disease.
The US drug regulator’s approval follows the end-stage trials which showed the drug Fabhalta (iptacopan), achieved a 44% proteinuria reduction in patients with primary immunoglobulin A nephropathy, or IgAN, compared with a 9% in the dummy group, according to a statement from Novartis.
The Food and Drug Administration’s approval marks the first such from Novartis’ renal pipeline, which also includes atrasentan and zigakibart for the treatment of IgAN.
“Today’s approval of Fabhalta as a first-in-class medicine for IgA nephropathy is an important milestone in our journey to evolve rare renal disease care by bringing new treatments to people in urgent need of options,” said Victor Bultó, President US, Novartis.
Albuminuria
Proteinuria, also called albuminuria, is elevated protein in the urine. It is not a disease in and of itself but a symptom of certain conditions affecting the kidneys, according to Johns Hopkins Medicine.
Too much protein in the urine means that the kidneys’ filters — the glomeruli — are not working properly and are allowing too much protein to escape into the urine.
When the glomeruli are damaged the condition is called nephritis or glomerulonephritis. Other conditions can lead to nephritis, including hypertension, heart disease and diabetes, as well as other types of kidney disease.
Fabhalta’s continued approval may be contingent on a continuing end-stage study evaluating whether the treatment slows disease progression, data from which is expected to be released next year.
IgAN is a progressive, rare disease in which the immune system attacks the kidneys, often causing glomerular inflammation and proteinuria. About 25 people per million worldwide are newly diagnosed with IgAN each year, according to Novartis. It mainly affects young adults.
Lose kidney function
In IgAN a protein called immunoglobulin A (IgA) becomes trapped in the very fine filters of the kidney (glomeruli), causing damage and scarring to the whole kidney, according to Kidney Research UK.
IgA is normally present in the bloodstream and its main role is to fight infections throughout the body. But in IgAN the body’s immune cells produce abnormally formed IgA.
Around 30% of IgAN patients will go on to lose kidney function and will require a transplant or life on dialysis. There are currently no reliable tests available to predict which of these patients, known as progressors, will lose their kidney function.
“The heterogeneous and progressive nature of IgA nephropathy has made it challenging to effectively treat this disease. Thankfully, the treatment landscape is rapidly evolving,” said Professor Dana Rizk, investigator of the study and professor at the University of Alabama at Birmingham Division of Nephrology.
Despite the current standard of care, up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis.
Continued evaluation
These patients often require maintenance dialysis or kidney transplantation. Effective, targeted therapies with different mechanisms of action can help physicians select the most appropriate treatment for patients.
Novartis stated that it wasn’t established whether Fabhalta slows kidney function decline in patients with IgAN.
The continued approval of Fabhalta may be contingent upon verification and description of clinical benefit from the ongoing end-stage study, evaluating whether the drug slows disease progression as measured by estimated glomerular filtration rate decline over 24 months.
“As a parent of a son living with the disease for 20 years, I understand firsthand the fear and uncertainty that comes with an IgAN diagnosis and the devastating impact it can have on patients and their families,” said Bonnie Schneider, Director and Co-Founder, IgAN Foundation.
“Today’s approval offers new hope for people living with IgA nephropathy as it represents a treatment innovation that provides us with a new way to fight this multifaceted disease.”
