After a rough few months for Intercept and Advanz Pharma's Ocaliva on both sides of the Atlantic, the other shoe has dropped for the rare liver disease drug in Europe.
The General Court of the European Union last week decided not to extend a suspension of the European Commission’s (EC’s) decision to revoke Ocaliva’s marketing authorization. Advanz Pharma, which holds commercial rights to the drug outside the U.S., shared the update in a press release.
Late this summer, the EC sought to pull Ocaliva’s approval in EU member states as well as Iceland, Liechtenstein and Norway following a reassessment of the drug’s benefit-risk profile. However, Intercept and Advanz were spared from the fallout—at least temporarily—when Europe’s General Court put the EC’s decision on hold.
The drug, which is also approved in the U.S. under the FDA’s accelerated pathway, is a second-line treatment for the autoimmune disease primary biliary cholangitis (PBC), which causes the liver’s bile ducts to become inflamed. The condition can lead to cirrhosis, liver transplant or death if left untreated.
In light of the suspension being lifted, Ocaliva’s European marketing nod has been revoked “with immediate effect,” Advanz said in its release.
Advanz’s CEO, Steffen Wagner, said in a statement that the company is “very disappointed” and disagrees with the court and the EC’s decision.
“Without Ocaliva, patients could be at increased risk of disease progression, including serious liver harm, liver transplantation or death,” Wagner said, adding that Advanz is currently in talks with relevant national agencies to ensure continued access to Ocaliva for patients who rely on the drug in Europe.
On that front, the European Medicines Agency has hinted at the potential for Advanz to continue providing Ocaliva to European patients on a compassionate access or named patient program basis, according to the company.
The situation for Ocaliva hasn’t been much better in the U.S., where the FDA last month shot down Intercept’s bid to convert an accelerated nod for the drug into a full approval.
The FDA’s rebuff followed a poor showing for the medicine at a September meeting of the FDA’s Gastrointestinal Drug Advisory Committee, during which a panel of outside experts largely voted against the prospect of a traditional green light.
The experts weighed Ocaliva’s clinical benefit and overall risk-benefit profile as depicted in two Intercept post-marketing trials.
The bright spot for Intercept is that Ocaliva will remain available in the U.S. for certain PBC patients under the accelerated approval it received back in 2016. The New Jersey-based drugmaker has also said it will work closely with the FDA on next steps for the drug, including additional evidence generation.
Separately, Intercept's attempts to win approval for Ocaliva to treat metabolic dysfunction-associated steatohepatitis failed to yield success on multiple occasions.