Nefecon filed with EMA for primary IgA Nephropathy – Calliditas Therapeutics

Calliditas Therapeutics has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for Nefecon (budesonide), a novel oral formulation targeting down regulation of IgA1 for the treatment of primary IgA Nephropathy (IgAN). The submission is based on positive data from Part A of the NefIgArd pivotal Phase III 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.

The study achieved its primary endpoint of proteinuria reduction compared to placebo, and also showed a renal protective effect by way of stabilization of eGFR at 9 months. The submission also includes clinical data from the Phase II NEFIGAN trial, which met the same primary and secondary endpoint as the NefIgArd study. The NefIgArd trial also showed that Nefecon was generally well-tolerated, with a safety profile in keeping with the Phase IIb results. 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.

The MAA for Nefecon has been granted Accelerated Assessment procedure by EMA’s Committee for Human Medicinal Products (CHMP), which is intended to expedite access to drugs that the CHMP considers to be of major therapeutic interest from the point of view of public health and in particular from the viewpoint of therapeutic innovation. Accelerated assessment reduces the maximum timeframe for review of the MAA to 150 days (excluding clock-stops). If approved, Nefecon could be available to patients in Europe in H1 2022 and would become the first therapy specifically designed and approved for the treatment of IgAN, and which has the potential to be disease modifying.