On Dec. 15, 2023, CymaBay Therapeutics announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for seladelpar for the management of primary biliary cholangitis (PBC). Seeking a Priority Review, the biopharmaceutical company anticipates that the FDA, upon acceptance, will conclude its evaluation within six months.
The NDA for seladelpar also lists it as a treatment for symptoms related to PBC, including pruritus (itching) in adults without cirrhosis or with compensated cirrhosis who incompletely responded or were intolerant to ursodeoxycholic acid.
Read more about PBC signs and symptoms
Efficacy and tolerability data from more than 500 patients with PBC in the placebo-controlled phase 3 RESPONSE and ENHANCE studies, the long-term, open-label ASSURE study and prior phase 2 studies provided the framework for the NDA application.
Klara Dickinson, Chief Regulatory and Compliance Officer, CymaBay Therapeutics, called the NDA submission an important milestone for the company, adding that her team will continue to work with the FDA to ensure validation of the application.
Researchers have found that seladelpar, characterized as a first-in-class oral, selective peroxisome proliferator-activated receptor delta (PPARδ) agonist, not only regulates critical metabolic and liver disease pathways but also the genes involved in bile acid synthesis, inflammation, fibrosis and lipid metabolism, storage, and transport.
It previously received Breakthrough Therapy Designation, which is granted by the FDA to investigational agents that have demonstrated the potential to confer substantial improvement over existing therapies. In October, the FDA updated this designation, citing data indicative of improved pruritus and alkaline phosphatase levels for patients without cirrhosis or with compensated cirrhosis.
Of note, seladelpar is the only agent of its kind to have demonstrated a statistically significant improvement in markers of cholestasis (impaired bile flow) related to the risk of disease progression and PBC-related pruritus in the phase 3 setting.
Per the press release, in the first half of 2024, CymaBay Therapeutics intends to file marketing authorization applications to the European Medicines Agency (EMA) and U.K. Medicines and Healthcare products Regulatory Agency (MHRA). The EMA has already granted Priority Medicines (PRIME) status to seladelpar, aiming for earlier patient access.
