CymaBay Therapeutics has filed a new drug application (NDA) with the US Food and Drug Administration (FDA) seeking approval for its investigational therapy, seladelpar, for primary biliary cholangitis (PBC) management.
Seladelpar is intended to manage PBC including pruritus in adult patients without cirrhosis or with compensated cirrhosis (Child-Pugh A) who did not respond to or are not tolerant of ursodeoxycholic acid.
CymaBay has sought a priority review of the application from the FDA.
If priority review is granted for seladelpar for this indication, the US regulator will conclude the process within six months of receiving the NDA.
Seladelpar has previously received breakthrough therapy designation from the FDA.
The application is based on data from placebo-controlled Phase III RESPONSE and ENHANCE clinical trials that assessed the efficacy and tolerability profiles of the therapy in more than 500 PBC patients.
The comprehensive data also includes findings from the long-term, open-label ASSURE trial and previous Phase II studies.
According to findings from a Phase III trial, seladelpar treatment offered a statistically significant improvement in cholestasis markers linked to progression risk and pruritus linked to PBC.
CymaBay Therapeutics chief regulatory and compliance officer Klara Dickinson stated: “People living with PBC need new treatment options to reduce the risk of disease progression and the daily impact of their disease from debilitating symptoms like pruritus.
“The announcement of our submission of the NDA for seladelpar is an important milestone in our ongoing work to bring forward new innovative therapies with the potential to help people living with PBC.
“We now look forward to working with the FDA to secure validation of the application and throughout its review.”
The company plans to submit marketing authorisation applications with the European Medicines Agency and the UK Medicines and Healthcare products Regulatory Agency in the first half of 2024.
The EMA has granted a priority medicines designation for seladelpar.
