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Promising Results from RESPONSE Trial: Seladelpar Significantly Effective in Primary Biliary Cholangitis

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Mason Walker
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Promising Results from RESPONSE Trial: Seladelpar Significantly Effective in Primary Biliary Cholangitis

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Primary Biliary Cholangitis (PBC), a chronic liver disease, has been the focus of several studies, with efforts to find improved treatments for patients who respond inadequately to conventional therapies. Among these is the RESPONSE phase 3 trial evaluating seladelpar, an investigational agent that has shown impressive results in treating PBC.

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RESPONSE Phase 3 Trial: Seladelpar vs Placebo

Published in The New England Journal of Medicine, the RESPONSE phase 3 trial measured the efficacy of seladelpar on adults diagnosed with PBC. The trial's primary endpoint was a biochemical response, defined as a reduction in alkaline phosphatase level with normal bilirubin. The trial involved patients who had either an inadequate response to or unacceptable side effects from ursodeoxycholic acid.

The trial results showed that a much larger percentage of patients treated with seladelpar met the primary endpoint compared with those treated with a placebo. Specifically, 61.7% of patients treated with seladelpar achieved the primary endpoint at Month 12, compared with 20.0% of patients treated with placebo. Moreover, the key secondary endpoint of alkaline phosphatase normalization was reached in 25.0% of patients treated with seladelpar, versus 0.0% for patients treated with placebo.

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Reduction in Pruritus and Liver Injury

Besides meeting the primary endpoint, seladelpar also demonstrated significant reductions in pruritus (itching) across the numerical rating scale and in the PBC 40 questionnaire. Patients reported a statistically significant reduction in pruritus at Month 6 and at Month 12. This reduction in pruritus provides a relief from one of the most distressing symptoms of PBC.

In addition, seladelpar showed rapid and sustained improvements in reducing cholestasis and liver injury. Cholestasis, a condition where bile flow from the liver to the gallbladder is slowed or stopped, is a common issue in PBC patients. By reducing cholestasis, seladelpar helps to alleviate the symptoms and slow the progression of the disease.

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Safety of Seladelpar

The safety of seladelpar, as reflected in adverse events, was similar in both the seladelpar-treated and placebo-treated groups. This finding suggests that seladelpar is not only effective but also safe for use in PBC patients.

Future of Seladelpar

Following the promising results from the RESPONSE trial, the FDA has accepted the New Drug Application (NDA) for seladelpar and granted it priority review. The FDA set a target action date of August 14, 2024. This acceptance is a testament to the potential of seladelpar as a promising treatment for PBC.

Overall, the results of the RESPONSE trial suggest that seladelpar could be a significant step forward in the treatment of PBC. With its demonstrated efficacy in reducing alkaline phosphatase levels and pruritus, along with its safety profile, seladelpar could potentially improve the lives of many PBC patients in the near future.

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