Nefecon filed for accelerated approval with FDA for primary IgA Nephropathy.- Calliditas Therapeutics

Calliditas Therapeutics announced the submission of a New Drug Application (NDA) to the FDA for Nefecon (budesonide), a novel oral formulation targeting down regulation of IgA1 for the treatment of primary IgA Nephropathy (IgAN). Calliditas is seeking accelerated approval under Subpart H for the 505(b)(2) application.The NDA submission is based on positive data from Part A of the NefIgArd pivotal Phase 3 study, a randomized, double-blind, placebo-controlled, international multicenter study designed to evaluate the efficacy and safety of Nefecon compared to placebo in 200 adult patients with IgAN. As previously reported, the study achieved its primary endpoint of proteinuria reduction compared to placebo, as well as showing stabilisation of eGFR at 9 months. The NefIgArd trial also showed that Nefecon was generally well-tolerated with a safety profile in keeping with the Phase 2b results. The submission also includes clinical data from the Phase II NEFIGAN trial, which also met the same primary and secondary endpoints as the NefIgArd study. Calliditas is the only company which has achieved positive data in randomized, double-blind, placebo-controlled Phase IIb and Phase III clinical trials in IgAN. Calliditas has applied for accelerated approval, which allows drugs targeting serious conditions that fill an unmet medical need to be approved based on a surrogate endpoint. The surrogate endpoint in the pivotal Phase III trial NefIgArd was reduction of proteinuria versus placebo, which is supported by the statistical framework based on the meta-analysis of clinical studies where an intervention was carried out in patients with IgAN, as updated by Thompson A et al, published in 20191. A confirmatory study designed to provide data on long-term renal benefit is fully recruited and is expected to read out in early 2023.