19 Deaths, Serious Liver Injury Linked to Intercept Pharma’s Ocaliva


September 22, 2017
By Alex Keown, BioSpace.com Breaking News Staff

NEW YORK – The U.S. Food and Drug Administration is warning health care providers to stick to dosing recommendations for Intercept Pharma (ICPT)’s liver disease medicine Ocaliva after 19 patient deaths were reported. Some of the patients were known to have been taking a higher dose than prescribed

Ocaliva was approved in October 2016 to treat the chronic liver disease known as primary biliary cholangitis. Of the 19 who died, the FDA said it received information about cause of death from eight patients. In seven of those cases, the cause of death was reported to be worsening of primary biliary cholangitis. The other case identified cardiovascular issues, the FDA said.

“These patients are receiving excessive dosing, particularly a higher frequency of dosing than is recommended in the drug label for them… The recommended dosing and monitoring for patients on Ocaliva are described in the current drug label. We are working with the drug manufacturer, Intercept Pharmaceuticals, to address these safety concerns,” the FDA said in its statement.

Primary biliary cholangitis causes the bile ducts in the liver to become inflamed, damaged and destroyed. The damage to the ducts leads to a buildup in bile, which damages the liver over time and eventually leads to the liver’s inability to function.

The FDA said the Ocaliva patients were taking the medication more frequently than the prescribed dosing recommendation. Seven of the patients were taking 5mg of Ocaliva every day instead of a dose “no greater than 10 mg twice weekly as recommended in the label prescribing information for patients with this extent of decreased liver function,” the FDA said.

Shares of Intercept fell more than 10 percent Thursday and are down more than 7 percent in pre-market trading.

Intercept has not commented on the FDA’s note, Bloomberg reported. However, the FDA’s announcement came two weeks after Intercept offered care providers prescriber information about Ocaliva, a farnesoid X receptor. The company confirmed there were issues of death and liver injury due to some patients taking the medicine more often than approved recommendations. In its note to healthcare providers, Intercept underscored the prescribing doses. Additionally, the company said patients should be monitored closely for adverse reactions. Intercept also said healthcare providers should “exercise a low threshold for drug discontinuation or interruption” based on toxicity results.

The news could be troubling for Intercept which had its eyes on Ocaliva becoming a blockbuster. Leerink analyst Joseph Schwartz said the FDA may resort to a black box warning for the drug due to the fatalities and the severe liver damage, according to Reuters.

However, RBC Capital Market analysts have a different take, Reuters said. The concerns about Ocaliva were already known and included on the drug’s prescribing label. More so, the patients who were affected by the problems cited by the FDA represent less than 5 percent of the overall PBC population. That means it would be likely any negative commercial impact would be too serious, the analysts said, according to Reuters. Approximately 15,000 prescriptions for Ocaliva have been written since it was approved, Bloomberg noted.


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