Primary immunoglobulin A nephropathy (IgAN) is a rare, progressive autoimmune disease that attacks the kidneys, most often developing between a person’s late teens and late 30s. On December 20, 2023, Stockholm-based Calliditas Therapeutics AB announced that the U.S. Food and Drug Administration (FDA) had approved a new drug called TARPEYO (budesonide), a delayed-release capsule designed to reduce kidney function loss in adults living with IgAN. The approval is based on Calliditas’ Phase 3 NeflgArd clinical trial. Learn about TARPEYO, how it works, and what it could mean for your treatment and quality of life.* 

About the research

The NefIgArd study was a randomized, double-blind, multicenter study involving 364 adult IgAN patients at risk for disease progression. To evaluate the efficacy and safety of TARPEYO, which was developed to specifically target an underlying cause of IgAN:

• Patients were randomized to receive either 16 mg/day oral capsules of TARPEYO or a matching placebo for 9 months, followed by a 15-month observation period without the study drug. 
• Patients were given TARPEYO or the placebo in addition to optimized renin angiotensin system inhibitors (RASi). RASi are given to people with chronic kidney disease to help manage hypertension, a key risk factor and complication of the disease. 

What they found

The study findings showed that, at two years, use of TARPEYO resulted in:

• A 6.11 decline in eGFR (percentage of kidney function), compared with a 12.0 decline in the placebo group
• A significant, and sustained, reduction in proteinuria (protein in urine, an indicator of kidney function decline)

Between 6% and 17% of participants who received TARPEYO experienced side effects that included: hypertension, swelling, muscle spasms, dermatitis, headache, weight gain, indigestion, joint pain, upper respiratory tract infection, and/or increased white blood cell count.

“The evidence of sustained reductions in proteinuria and a clinically significant reduction in the loss of eGFR, which can help slow the progression towards dialysis or transplant care, highlights the potential of TARPEYO as a disease-modifying agent,” said said Richard Lafayette, M.D., FACP, Stanford Healthcare. 

What it means

“This first-ever IgAN treatment to get a full approval based on kidney function represents a beacon of hope for the entire IgA nephropathy community and signifies a critical step forward in the battle against IgAN,” said Bonnie Schneider, director and cofounder of the IgAN Foundation.

TARPEYO is currently available only through Calliditas’ specialty pharmacy, Biologics by McKesson. With the intention of preventing cost from being a barrier to care, the pharmacy provides an enrollment form that can connect patients to various benefits, including financial aid programs. 

“We are thrilled that adult IgAN patients at risk for progression in the United States can now have access to this pioneering treatment option,” said Renee Aguiar-Lucander, CEO of Calliditas. “I am incredibly proud of the team’s unwavering commitment to the goal of preventing end-stage renal disease in patients with this challenging rare disease.”

*Calliditas Therapeutics. (2023, December 2023). Calliditas Therapeutics announces full FDA approval of TARPEYO®, the only FDA-approved treatment for IgA nephropathy to significantly reduce the loss of kidney function. https://www.calliditas.se/en/calliditas-therapeutics-announces-full-fda-approval-of-tarpeyo-the-only-fda-approved-treatment-for-iga-nephropathy-to-significantly-reduce-the-loss-of-kidney-function/

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