The court sided with the EU in the decision to cancel Ocaliva’s license

Advanz Pharma’s bid to keep Ocaliva treatment for the rare liver disease primary biliary cholangitis (PBC) on the EU market has failed.

European Commission canceled Conditional marketing authorization for Okaliva (obeticholic acid) earlier this year for the treatment of PBC, a rare and progressive autoimmune disease that mainly affects women, resulting from the destruction of the bile ducts in the liver, on the grounds that its benefits no longer outweigh its risks.

Advanz won provisionally to delay In September, after the General Court of the European Union ruled to suspend 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 Ocaliva can no longer be offered to PBC patients in EU member states and EEA countries (Iceland, Liechtenstein and Norway) with immediate effect.

According to the European Association for the Study of the Liver (EASL), if they do not respond to or cannot tolerate the standard first-line treatment, ursodeoxycholic acid (UDCA), patients are limited to second-line treatment options. We are lobbying to keep Ocaliva available to patients who cannot tolerate or respond to UDCA treatment.

Until very recently, there were no second-line options for PBC patients in the EU other than Ocaliva; However, this changed in September when Ipsen’s PPAR agonist Iqirvo was approved by the Commission in combination with UDCA. As monotherapy in adults who do not respond to UDCA alone and in those who cannot tolerate the drug.

Another potential option is Gilead Sciences PPAR agonist Livdelzi (seladelpar), a case submitted to the EU by Gilead subsidiary CymaBay Therapeutics earlier this year and is being reviewed by the EMA.

In a statement, Advanz’s CEO, Steffen Wagner, objected to the court’s new decision, saying that this decision only measured whether there had been “serious” and “irreparable” or “irreversible” harm to the company by the EC decision, and that he did not oppose this decision. to assess the impact on patients or the validity of the Commission’s interpretation.

“Ocaliva is a much-needed treatment option for thousands of PBC patients in Europe,” he said.

“As an FXR agonist, (it) has a different mechanism of action than other treatments and is supported by ample positive real-world evidence from over seven years of clinical use. Without Ocaliva, patients may be at increased risk.” Disease progression, including severe liver damage, liver transplantation, or death.”

The only options open to existing patients who want continued access to Ocaliva are to obtain it through the compassionate use or named patient program, and Advanz said it is exploring how to keep that path open.

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