Affecting approximately 100,000 people in the US, PBC is a rare, autoimmune, cholestatic liver disease that occurs when a buildup of bile and toxins, along with chronic inflammation, causes irreversible scarring of the liver and destruction of the bile ducts.
If left untreated, the lifelong condition can progress, leading to liver failure and potentially the need for a liver transplant.
The FDA’s decision was based on results from the phase 3 ELATIVE trial, published in the New England Journal of Medicine, which demonstrated that 13 times more patients achieved the composite primary endpoint of biochemical response when treated with Iqirvo plus UDCA compared to placebo plus UDCA.
Secondary endpoints demonstrated normalisation in alkaline phosphatase (ALP), a biochemical marker used as a surrogate endpoint in PBC trials, levels in 15% of Iqirvo-treated patients versus 0% for placebo plus UDCA.
Marked as “the first new medicine for PBC in nearly a decade”, Christelle Huguet, executive vice president, head of research and development, Ipsen, added: “Iqirvo demonstrated statistically significant improvements in biochemical response compared to UDCA alone [and] is therefore a much-needed treatment option.”
Dr Kris Kowdley, director, Liver Institute Northwest, Washington and primary investigator on the study, said: “The approval of Iqirvo will allow healthcare providers in the US to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC.”
Iqirvo was discovered and developed by Genfit and Ipsen licensed the exclusive worldwide rights (except in China, Hong Kong, Taiwan and Macau) to the drug in 2021.
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