Calliditas Therapeutics has announced that the U.S. Food and Drug Administration has accepted the submission and granted Priority Review for the New Drug Application (NDA) for Nefecon, a down regulator of IgA1 for the treatment of IgA nephropathy (IgAN.)
The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of September 15, 2021, states the company in a press release.
“We are very excited about being granted priority review, which reflects the unmet medical need of IgAN. We look forward to engaging with the agency and work towards an accelerated approval later this year so that we will be in a position to provide the first approved medication for IgAN patients,” says CEO Renée Aguiar-Lucander.
Prepare for commercialization
Calliditas filed an NDA with the FDA on March 15, 2021. The NDA submission is based on positive data from Part A of the NefIgArd 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. The NefIgArd study read out data in November 2020 and achieved its primary endpoint of proteinuria reduction compared to placebo as well as showing stabilization of eGFR at 9 months. 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. Both studies showed that Nefecon was generally well-tolerated, with a similar safety profile across both sets of results.
“Having a target action date provides us with a clear timeline as we continue to expand our US organization and prepare for commercialization in the fourth quarter of this year, subject to approval,” said Head of North America Commercial Andrew Udell.
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 Phase 3 trial NefIgArd was reduction of proteinuria versus placebo. The confirmatory Part B of the NefIgArd study, designed to provide data on long-term renal benefit, is fully recruited and is expected to read out in early 2023, states the company.
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.
Photo of Renée Aguiar-Lucander, CEO, Calliditas: Jann Lipka