Pharma Focus Europe

CymaBay Submits New Drug Application to FDA for Approval of Seladelpar in Primary Biliary Cholangitis Treatment

Saturday, December 16, 2023

CymaBay Therapeutics, Inc. (NASDAQ: CBAY) has achieved a notable milestone in advancing innovative therapies for liver and chronic diseases. The biopharmaceutical company has officially submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for seladelpar, an investigational treatment designed to manage primary biliary cholangitis (PBC) in adults.

Seladelpar, characterized as a potent and selective peroxisome proliferator-activated receptor delta (PPARδ) agonist, received Breakthrough Therapy Designation from the FDA. Results from Phase 3 trials have demonstrated a significant improvement in markers of cholestasis and PBC-related pruritus. The NDA, seeking approval for use in adults who have not adequately responded to or cannot tolerate ursodeoxycholic acid (UDCA), comes with a request for Priority Review. If accepted, this could lead to an FDA decision within a six-month timeframe.

The application draws support from a robust dataset originating from the placebo-controlled Phase 3 RESPONSE and ENHANCE studies, the long-term open-label ASSURE study, and earlier Phase 2 studies, involving over 500 participants with PBC. Breakthrough Therapy Designation is granted when preliminary evidence indicates substantial improvement over existing therapies, and seladelpar's designation was updated in October 2023 based on promising data.

CymaBay is also gearing up to submit marketing authorization applications to the European Medicines Agency (EMA) and U.K. Medicines and Healthcare products Regulatory Agency (MHRA) in the first half of 2024. Seladelpar has secured Priority Medicines (PRIME) status from the EMA, recognizing its potential to address unmet medical needs and expedite patient access.

PBC, a rare inflammatory liver disease primarily affecting women, poses a significant health risk, and there is a pressing need for new treatment options. The potential approval of seladelpar could offer relief for patients by addressing symptoms such as pruritus and reducing the risk of disease progression. CymaBay expresses gratitude to participants, their families, and investigators involved in seladelpar studies and looks forward to collaborating with the FDA during the review process.



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