The European Medicines Agency (EMA) has validated and accepted for review the Marketing Authorization Application (MAA) for seladelpar for the treatment of primary biliary cholangitis (PBC), including pruritus, in certain patients who have not responded to treatment with ursodeoxycholic acid, according to a press release from CymaBay Therapeutics, the manufacturer of the therapy.
Seladelpar is an investigational, potent, selective peroxisome proliferator-activated receptor delta agonist that is currently being developed for the treatment of adult patients with PBC. The MAA contains data from the seladelpar PBC clinical development program in >500 individuals with PBC, which includes findings from the phase 3 RESPONSE trial.
Findings from RESPONSE were recently published in The New England Journal of Medicine. The primary endpoint measure in the study was defined as an alkaline phosphatase level of <1.67 times the upper limit of the normal range, with a decrease of ≥15% from baseline and a normal total bilirubin level at 12 months. This primary endpoint is reflective of registrational studies of the current second-line PBC therapy that has been authorized by the EMA.
In RESPONSE, seladelpar treatment showed statistically significant improvements in biochemical markers of disease progression and PBC-associated pruritus prespecified endpoints. Rates of adverse events reported were similar in the seladelpar and the placebo treatment groups.
Read more about PBC etiology
According to Klara Dickinson, Chief Regulatory and Compliance Officer of CBAY, “People living with PBC in Europe today have limited treatment options and can face both disease progression and a lifetime of symptoms[, such as] itch and fatigue[,] that can significantly impact their quality of life.” Ms. Dickinson emphasized, “New treatment options are needed.”
Under the umbrella of the European centralized licensing procedure, the EMA Committee for Medicinal Products for Human Use will review the MAA for all 27 members of the European Union, along with Iceland, Liechtenstein and Norway.
In 2016, seladelpar received Priority Medicines (PRIME) status from the EMA, as part of “its program to enhance support for the development of medicines that target an unmet need.” The agent has been accepted for review by the UK Medicines and Healthcare products Regulatory Agency. Moreover, the US Food and Drug Administration has accepted a New Drug Application for priority review of seladelpar.
A rare, chronic inflammatory hepatic disorder, PBC can lead to liver cirrhosis and an elevated risk for liver-associated mortality. New treatments aim to prevent additional disease progression by decreasing inflammation and bile acids in the liver, as well as providing meaningful relief of pruritus.
“Validation of the European Marketing Authorization Application for seladelpar and the start of the agency’s formal review is an important step forward in seladelpar’s journey to potential approval in Europe. We look forward to further discussion with the agency and rapporteurs as they conduct their review,” Dickinson stated.
