CymaBay has requested a Priority Review of the NDA that, if accepted, would mean the FDA would plan to complete its review within six months of accepting the application.
Seladelpar has been granted Breakthrough Therapy Designation by the FDA and is the only potent, selective, orally active delpar, or PPAR agonist, with Phase 3 trial results demonstrating a statistically significant improvement in markers of cholestasis related to risk of progression and PBC-related pruritus. The comprehensive NDA application is supported by robust data evaluating the efficacy and tolerability profile of seladelpar in more than 500 participants with PBC in the placebo-controlled Phase 3 RESPONSE and ENHANCE studies, the long-term open-label ASSURE study, as well as in prior Phase 2 studies.
Breakthrough Therapy Designation is granted by the FDA to investigational agents intended to treat a serious condition and preliminary clinical evidence indicates that it may demonstrate substantial improvement over available therapy on a clinically significant endpoint. FDA updated the Breakthrough Therapy Designation for seladelpar in
'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. Today's 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,' said
CymaBay intends to file marketing authorization applications to the
About PBC
PBC is a rare, chronic inflammatory liver disease primarily affecting women (1 in 1,000 women over the age of 40 or about 130,000 total people in the
About Seladelpar
Seladelpar, an investigational treatment for people with PBC, is a first-in-class oral, selective peroxisome proliferator-activated receptor delta (PPAR) agonist, or delpar, shown to regulate critical metabolic and liver disease pathways in indications with high unmet medical need. Preclinical and clinical data support its ability to regulate genes involved in bile acid synthesis, inflammation, fibrosis and lipid metabolism, storage, and transport.
About CymaBay
Cautionary Statements
Any statements made in this press release regarding potential FDA acceptance of the seladelpar NDA, its subsequent review and the timing thereof, the potential for seladelpar to treat PBC and potentially improve ALP levels, clinical symptoms or outcomes of the disease, the future EMA and MHRA filing plans of CymaBay and the timing thereof are forward-looking statements that are subject to risks and uncertainties. Actual results and the timing of events regarding the further development of seladelpar could differ materially from those anticipated in such forward-looking statements as a result of risks and uncertainties, which include, without limitation, risks related to: the success, cost and timing of any of CymaBay's product development activities, including clinical trials and effects observed in trials to date that may not be repeated in the future. Additional risks relating to CymaBay are contained in CymaBay's filings with the
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