US FDA staff questions confirmatory trial data for Intercept's liver disease drug
Rewrites paragraph 1, adds details from FDA and company's briefing documents in paragraphs 7, 8 and 9 and background in paragraphs 10 and 11
Sept 11 (Reuters) - Staff reviewers at the U.S. Food and Drug Administration said on Wednesday a confirmatory trial did not show the effectiveness of Intercept's liver disease drug, which received the health regulator's accelerated nod in 2016.
Under the accelerated pathway, companies are required to conduct additional trials to establish their drug's benefits. Failure to do so can sometimes lead to market withdrawal.
Intercept's oral drug, Ocaliva, had been approved under the accelerated approval pathway to treat patients with primary biliary cholangitis (PBC), a rare disease that causes inflammation of small bile ducts in the liver and can eventually destroy them.
In 2021, FDA restricteduse of Ocaliva to PBC patients who did not have advanced cirrhosis or severe scarring of the liver.
The FDA had said some patients with advanced cirrhosis who received Ocaliva developed liver failure.
The staff advisers said on Wednesday Ocaliva'sconfirmatory trial did not show the drug waseffective in PBC patients with or without advanced scarring.
They also raised concerns about the drug's safety, as 11 patients who received it during the trial died or required a liver transplant, compared with two patients on placebo.
The concerns come ahead of a meeting of independent experts on Friday, where panelists will vote on whether the drug's benefits outweigh its risks.
Intercept said only one case of liver transplant was possibly related to the drug.
The company, which holds the drug's U.S. commercialrights, was acquired by Italy's Alfasigma fornearly $800 million last year. Intercept had reported sales of $152 million for the first half of 2023.
The FDA last month granted accelerated approval to Gilead's GILD.O drug for treating PBC, Livdelzi.
Accelerated approvals allow FDA to move drugs for life-threatening conditions to the market more quickly, but they have been criticized because some drugs are later proven to be ineffective.
Reporting by Bhanvi Satija in Bengaluru; Editing by Shailesh Kuber and Shreya Biswas
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