Calliditas Therapeutics, a leading pharmaceutical company, has exciting news to share. The U.S. Food and Drug Administration (FDA) has accepted the submission for the supplemental New Drug Application (sNDA) for Tarpeyo (budesonide) delayed release capsules, and in a significant stride, the application has been granted Priority Review. Mark your calendars for the Prescription Drug User Fee Act (PDUFA) goal date set for December 20, 2023.
“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. 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 Calliditas
Tarpeyo has already secured accelerated approval for its role in reducing proteinuria in adults facing the challenges of primary IgA nephropathy (IgAN) and are at risk of rapid disease progression. This typically includes individuals with a urine protein-to-creatinine ratio (UPCR) ≥1.5g/g.
The driving force behind this sNDA is the robust dataset stemming from the Phase 3 NefIgArd clinical trial. This rigorous, randomized, double-blind, multicenter study delved into the efficacy and safety of Tarpeyo (known as Nefecon® during development) at a daily dosage of 16 mg, compared to placebo. The trial focused on adult patients contending with primary IgAN who were on optimized RASi (renin-angiotensin system inhibitor) therapy. The results of this trial were compelling, revealing a statistically significant advantage of Nefecon over placebo (with a remarkable p-value of < 0.0001) in terms of estimated glomerular filtration rate (eGFR) over a two-year period. This study encompassed nine months of active treatment with Nefecon or placebo, followed by a 15-month observation period off the study drug. The data illustrated the benefits of Nefecon across the entire study population, irrespective of UPCR baseline, and showcased a notable difference between Tarpeyo and placebo in the two-year eGFR total slope—approximately 3mL/min per year, determined through a robust regression analysis method.
“We take great pride in the strong clinical evidence we have gathered, which we believe demonstrates TARPEYO’s durable and clinically meaningful impact on kidney function in the treatment of IgAN. 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.”
– Richard Phillipson, Chief Medical Officer at Calliditas
Calliditas is also collaborating closely with its esteemed European commercial partner, STADA Arzneimettel AG, with the aim of securing full approval for Nefecon (which already holds conditional approval under the name Kinpeygo®) from the European Commission, encompassing the entire study population.
This strategic and clinical advancement marks another significant stride forward in the journey towards improving the lives of those grappling with the complexities of IgA nephropathy. Calliditas Therapeutics’ dedication to transformative pharmaceutical solutions is reaffirmed through this commendable achievement.
