FDA Sets PDUFA Date for Full Approval of Tarpeyo

The FDA has accepted the submission for the supplemental new drug application (sNDA) for Tarpeyo (budesonide) and granted the application priority review. The NDA is seeking full approval of Tarpeyo to reduce protein level in the urine of adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression. The Prescription Drug User Fee Act (PDUFA) goal date is Dec. 20, 2023.

Primary immunoglobulin A nephropathy (IgAN) is a rare, progressive, chronic autoimmune disease that attacks the kidneys. Also called Berger’s disease, it can lead to progressive kidney damage. IgAN most often develops between late teens and late 30s. About 60,000 in the United States have IgAN, according to the National Kidney Foundation.

Develped by Calliditas Therapeutics, Tarpeyo was granted accelerated approval in December 2021. It is an oral, delayed release formulation of budesonide, a corticosteroid. Tarpeyo 4 mg has a list price of about $15,934 for a supply of 120 capsules, according to Drugs.com. The recommended dose is 16 mg once daily.

Renee Aguiar-Lucander

The sNDA is based on the full data data set from the phase 3 NefIgArd clinical trial, which studied the once-daily dose of 16 mg, compared with placebo. The trial demonstrated a statistically significant benefit of Tarpeyo over placebo in estimated glomerular filtration rate (eGFR) over the two-year study period. The data reflected treatment benefits across the entire study population. The majority of treatment-emergent adverse events (TEAE) being mild or moderate, and with TEAEs leading to discontinuation of study drug in less than 10% of patients.

The full results of this studied were recently published in The Lancet.

“The significant eGFR treatment benefit observed across the entire study population provides further evidence that TARPEYO can be disease-modifying, potentially significantly delaying the need for dialysis or kidney transplantation for patients at risk,” Renee Aguiar-Lucander, CEO of Callidita, said in a press release.