Drugs

Ocaliva Confirmatory Trial Fails: FDA

FDA says a confirmatory trial for Intercept Pharmaceuticals’ liver disease drug Ocaliva (obeticholic acid), which received accelerated approval on 5/27/2016 to treat primary biliary cholangitis (PBC) in conjunction with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA or as monotherapy in adults unable to tolerate UDCA, did not confirm the drug’s effectiveness. The analysis by CDER medical reviewers is included in a briefing document released by FDA in advance of a 9/13 meeting of the agency’s Gastrointestinal Drugs Advisory Committee to consider Intercept’s submission of required postmarketing trial data.

In 2021, FDA restricted the use of the drug to PBC patients who did not have advanced cirrhosis or scarring of the liver, based on adverse events reported to FDA showing that some patients with advanced cirrhosis who received Ocaliva developed liver failure.

In addition to saying the confirmatory trial did not show the drug was effective in PBC patients with or without advanced scarring, the reviewers also voiced safety concerns as 11 patients who received the drug during the trial died or needed a liver transplant, compared to two patients who received a placebo.

The committee members will be asked to consider:

whether the results from the clinical outcomes trial and observational trial that Intercept submitted confirm the clinical benefits of Ocaliva in preventing hepatic decompensation, liver transplant, and death in the treatment of PBC;
whether, if doubt remains about clinical benefit, additional observational data or another randomized clinical trial would be needed to confirm clinical benefit; and
whether the available evidence demonstrates that the clinical benefit of Ocaliva outweighs the risks for treating PBC.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

Read more

Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome