Court sides with EU on decision to revoke Ocaliva's license

 Advanz Pharma's bid to keep its Ocaliva treatment for rare liver disease primary biliary cholangitis (PBC) on the EU market has ended in failure.

The European Commission revoked the conditional marketing authorisation for Ocaliva (obeticholic acid) earlier this year on the grounds that its benefits no longer outweighed its risks as a treatment for PBC, a rare and progressive autoimmune disease – mainly affecting women – that results from the destruction of the bile ducts in the liver.

Advanz won a temporary reprieve in September after the General Court of the European Union ordered a suspension of the EC's decision, allowing the FXR agonist to remain on sale, but the company confirmed this morning that the court had decided not to extend the suspension any further.

The new order means that Ocaliva can no longer be made available to PBC patients in EU member states, as well as EEA countries (Iceland, Liechtenstein, and Norway), with immediate effect.

That leaves patients with limited second-line options for treatment if they fail to respond to or cannot tolerate ursodeoxycholic acid (UDCA), the standard first-line therapy, according to the European Association for the Study of the Liver (EASL), which has been lobbying for Ocaliva to remain available for patients who cannot tolerate or do not respond to UDCA treatment.

Until very recently, there were no second-line options for patients with PBC other than Ocaliva in the EU, although, that changed in September when Ipsen's PPAR agonist Iqirvo was approved by the Commission in combination with UDCA in adults who don't respond to UDCA alone, and as a monotherapy for those who can't tolerate the drug.

Another potential option is Gilead Sciences PPAR agonist Livdelzi (seladelpar), which was filed in the EU earlier this year by Gilead's subsidiary CymaBay Therapeutics and is under review by the EMA. 

In a statement, Advanz's chief executive, Steffen Wagner, took issue with the court's new ruling, saying that it only gauged whether there were "serious" and "irreparable" or "irreversible" damages to the company by the EC decision, and did not assess the impact on patients or the validity of the Commission's interpretation.

"Ocaliva is a much-needed treatment option for thousands of patients with PBC in Europe," he remarked. 

"As an FXR agonist, [it] has a different mechanism of action from other treatments and is backed by a wealth of positive real-world evidence, gathered from more than seven years of clinical use. Without Ocaliva, patients could be at increased risk of disease progression, including serious liver harm, liver transplantation, or death."

The only option open for existing patients seeking continued access to Ocaliva now is to get it via a compassionate-use or named-patient programme, and Advanz said it is looking into how this avenue may be kept open.

Meanwhile, Ocaliva is also facing difficulty on the other side of the Atlantic, with the FDA declining to grant full approval to the drug in PBN earlier this month. It will however continue to be available to patients under its accelerated approval status, which was granted in 2016, according to Intercept Pharma, which holds US rights.

https://pharmaphorum.com/news/court-sides-eu-decision-revoke-ocalivas-license