CymaBay Therapeutics (CBAY) Announces FDA Acceptance of NDA and Priority Review for Seladelpar for the Treatment of Primary Biliary Cholangitis

February 12, 2024 8:45 AM EST

CymaBay Therapeutics, Inc. (NASDAQ: CBAY), a biopharmaceutical company focused on innovative therapies for patients with liver and other chronic diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted its New Drug Application (NDA) for seladelpar, an investigational treatment for the management of primary biliary cholangitis (PBC) including pruritus in adults without cirrhosis or with compensated cirrhosis (Child Pugh A) who are inadequate responders or intolerant to ursodeoxycholic acid. The FDA has granted priority review and set a Prescription Drug User Fee Act (PDUFA) target action date of August 14, 2024 and notified the company that it is not currently planning to hold an advisory committee meeting to discuss the application.

PBC is a rare, chronic condition that can lead to inflammation and eventually liver cirrhosis. Disease progression is associated with an increase in liver-related complications, including mortality. People living with PBC can also experience symptoms that significantly impact their quality of life such as pruritus (itch) and fatigue. New treatment options seek to help prevent further disease progression by reducing inflammation and bile acids in the liver and help provide meaningful relief of pruritus.

Seladelpar is an investigational, potent, selective, orally active PPARĪ“ agonist, or delpar, in development for PBC treatment. It is the only investigational agent to have demonstrated a statistically significant improvement in biochemical markers of disease progression and pre-specified measures of PBC-related pruritus in a Phase 3 study. The safety profile of seladelpar in the pivotal Phase 3 RESPONSE study, measured through rates of adverse events, was similar in those treated with seladelpar and placebo.

The NDA includes data from across the seladelpar PBC clinical development program in over 500 participants living with PBC, including from the placebo-controlled Phase 3 RESPONSE and ENHANCE studies, the long-term open-label ASSURE study, and prior Phase 2 studies. The FDA has previously granted seladelpar Breakthrough Therapy Designation (BTD) which enabled early submission of select non-clinical data through the rolling review process afforded by seladelpar's BTD status. FDA updated the Breakthrough Therapy Designation for seladelpar in October 2023 in recognition of clinical data that indicated seladelpar may provide meaningful improvement over existing therapy based on a reduction in alkaline phosphatase (ALP) and improvement in pruritus in patients without cirrhosis or with compensated cirrhosis.

"Acceptance of the seladelpar NDA for the treatment of PBC, is an important step forward in our ongoing mission to make this potential, novel, targeted treatment option available for people living with PBC," said Klara Dickinson, Chief Regulatory and Compliance Officer, CymaBay Therapeutics. "We are encouraged by the agency's decision to grant priority review for seladelpar, and its recognition of the significant need for new treatment options for people living with PBC. If approved, we believe seladelpar has the potential to raise the bar in second-line treatment of PBC by reducing markers of disease progression and improving pruritus, so we look forward to continued discussion with the agency throughout its review."

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