Positive budesonide trial results earn FDA drug application approval


Calliditas Therapeutics has announced that the US Food and Drug Administration (FDA) has accepted a submission for a supplemental New Drug Application (sNDA) for Tarpeyo (budesonide) delayed release capsules, and has granted priority review. The Prescription Drug User Fee Act (PDUFA) goal date is 20 December 2023.

Tarpeyo is currently approved under accelerated approval to reduce proteinuria in adults with primary IgA nephropathy (IgAN) at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5g/g.

“We are delighted to have been granted priority review by the FDA, which brings us one step closer to hopefully being able to provide TARPEYO to all patients at risk of progression and provide physicians with a tool to target the source of this disease to slow kidney function decline,” said Renee Aguiar-Lucander, CEO of Calliditas. “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.”

The sNDA is based on the full data set from the Phase 3 NefIgArd clinical trial, a randomised, double-blind, multicentre study evaluating the efficacy and safety of Tarpeyo (developed under the project name Nefecon) at a once-daily dose of 16mg, compared to placebo, in adult patients with primary IgAN on optimised RASi therapy.

The trial results, published in The Lancet, demonstrated a statistically significant benefit of Nefecon over placebo (p<0.0001) in estimated glomerular filtration rate (eGFR) over the two-year study period, which consisted of nine months of treatment with Nefecon or placebo, followed by a 15-month follow-up period off the study drug. The data reflected treatment benefits across the entire study population, regardless of UPCR baseline, and showed a difference between Tarpeyo and placebo in two-year eGFR total slope of approximately 3mL/min per year using a robust regression method of analysis.

Richard Phillipson, chief medical officer at Calliditas, stated: “The combination of the significant eGFR benefit and the reduction in proteinuria lasting for the entire 15-month follow-up period in the full results of our Phase 3 study provide strong rationale for establishing Tarpeyo as the standard of care for IgAN patients.”

Calliditas is also collaborating with its European commercial partner, STADA Arzneimettel AG, to seek full approval of Nefecon (which received conditional approval under the brand name Kinpeygo) by the European Commission in the full study population.



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