Calliditas Therapeutics AB (NASDAQ: CALT, Nasdaq Stockholm: CALTX) (“Calliditas”) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Nefecon, 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.
“This is a key milestone in the company’s development and we are looking forward to engaging with the agency. This is the first time a drug specifically designed for IgAN is being submitted for approval to the FDA and I believe that we are delivering a very robust data package based on the successful outcome of our pivotal Phase 3 trial and our large Phase 2b trial, which also met both the primary and key secondary endpoints. Calliditas has long been pioneering a treatment for IgAN based on precision and disease modification that focuses on the origin of the disease, with the hope of bringing help to thousands of patients, so today is truly a special day,” said CEO Renée Aguiar-Lucander.
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 2 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 2b and Phase 3 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 3 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.
If approved, Nefecon would become the first therapy specifically designed and approved for the treatment of IgAN, with the potential to be disease modifying. Subject to approval by the FDA, Calliditas intends to commercialize Nefecon for IgAN on its own in the United States.