Calliditas plans to file for full approval of Tarpeyo for kidney disease

CAR-T Digest - August 2020

Weekly Digest - 10-17 Mar 2023

March 13, 2023: Nefecon (Delayed-release Budesonide, Oral)/ IgA Nephropathy/ Calliditas: Plans to file for full approval post-Phase 3 data

Calliditas believes Phase 3 confirmatory data for Nefecon for primary IgA Nephropathy may aid in full approval from the FDA
- Nefecon, marketed as Tarpeyo, received accelerated approval from the FDA in December 2021 & conditional marketing authorization from the EU Commission in July 2022
The FDA approval will be based on the results of the Phase 3 trial:
- Statistically significant benefit over placebo in a 2-year period within 9 months of treatment
- Primary endpoint of eGFR with average 5.05 mL with Nefecon vs placebo (p<0.0001) over two years
Trial results were released shortly after Calliditas’ rival, Travere Therapeutics announced accelerated approval for Sparsentan.
- Confirmatory trial for Sparsentan is ongoing; topline results expected in Q4’23
Full approval from the FDA, EU Commission & UK’s MRA is expected in 2023
“This reflects the sustained impact on kidney function across the entire study population with a treatment which was specifically designed to treat IgAN by downregulating pathogenic IgA1 antibodies at their presumed source and we believe this dataset supports regulatory filing for full approval based on the Phase 3 study population,” said Renée Aguiar-Lucander, CEO of Calliditas
“The pattern of proteinuria change that we see with this drug is completely different to the pattern of proteinuria change we see with this high-dose systemic steroids are telling me that we are doing something fundamentally different in terms of the disease pathogenesis. And to see that the proteinuria is still at 30% lower at two years, despite only having a nine-month treatment, suggests to me that we are doing something fundamental to the production of the abnormal IGA,” said Johnathan Barrett, Professor of Renal Medicine, University of Leicester