Novartis received accelerated approval from the FDA for a treatment for IgA nephropathy, an autoimmune disease in which too many of an antibody build up in the kidney.
The drug, known as iptacopan, is marketed as Fabhalta. It was first approved in December for a rare blood disorder called paroxysmal nocturnal hemoglobinuria, or PNH, and costs $550,000 per year.
That cost, while comparable to other treatments for PNH, is substantially higher compared to other therapies for IgA nephropathy that have received accelerated approval in recent years, with Travere Therapeutics’ Filspari and Calliditas’ Tarpeyo priced at about $120,000 and $180,000 a year, respectively.
“We have considered the value of Fabhalta across all potential indications for which this innovative medicine is being studied,” Novartis wrote in an emailed statement. “Because Fabhalta is a complement inhibitor, we also took into consideration the price of comparable medicines in the same class currently on the market for various diseases.”
The accelerated approval was based on clinical trial data that showed Fabhalta reduced buildup of antibodies in patients’ urine significantly more than placebo at nine months. The treatment works via the complement pathway of the immune system. Novartis is working to produce confirmatory data to prove the drug slows kidney function decline over two years.
IgA nephropathy has become a more competitive area of new drug development. Vertex paid $4.9 billion to acquire Alpine Immune Sciences and its experimental IgA nephropathy drug and is planning to start a Phase 3 study later in August.
Novartis is also developing two other drugs for IgA nephropathy. It’s awaiting an FDA decision on an oral candidate, atrasentan, and is currently studying in Phase 3 an antibody treatment zigakibart, both of which it acquired in a $3.2 billion buyout of Chinook Therapeutics last year.
“The heterogeneity contributes to the need of effective targeted therapies with different mechanisms of action that can help prevent the progression of kidney failure,” said Victor Bulto, Novartis’ US president, in an interview ahead of the approval.
Both Vertex and Novartis hope their treatments will become a “pipeline-in-a-product” that can address a host of other immune system-related conditions. Novartis is developing Fabhalta in a range of rare diseases including lupus nephritis, and is planning to submit applications for approval in the US and EU for the drug in rare kidney conditions known collectively as C3 glomerulopathies by the end of the year.
In the second quarter of the year, Fabhalta generated $22 million in sales.