Ipsen announced that the European Commission has conditionally approved Iqirvo (elafibranor) 80mg tablets for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA).

Iqirvo is a first-in-class, oral, peroxisome proliferator-activated receptor (PPAR) agonist, that exerts an effect on the proteins PPARalpha and PPARo, thought to be key regulators of bile acid, inflammation and fibrosis.

“We are delighted that Iqirvo is approved in the E.U. as an effective new option for the treatment of people living with PBC. This is a rare liver disease, predominantly found in women, which hasn’t seen new innovation for nearly a decade. This is despite up to half of patients being intolerant or unresponsive to current therapies,” said Sandra Silvestri, Chief Medical Officer, Ipsen. “For those patients with PBC that may be at risk of disease progression and who continue to suffer from debilitating symptoms of the disease, we are delighted to be able to offer an effective treatment choice.”

Approval of Iqirvo was based on data from the phase III ELATIVE trial, which demonstrated a statistically significant treatment benefit with a 47% placebo-adjusted difference (P<0.001) between patients on Iqirvo 80mg (51%) compared with patients on placebo (4%) achieving a biochemical response. A greater decrease in PBC Worst Itch-NRS score from baseline was also observed for patients on Iqirvo versus placebo, but this was not statistically significant. Treatment with Iqirvo was associated with an improvement in pruritus (itch) as evidenced by a greater reduction in PBC-40 itch and 5-D itch total scores compared to placebo. Similar percentages of patients in the Iqirvo group and the placebo group experienced adverse events, treatment-related adverse events, severe or serious adverse events or adverse events leading to discontinuation.

“It is a positive development in the treatment and management of PBC that we have a new, efficacious and well-tolerated treatment for our patients living with the condition,” said Dr Marco Carbone, Professor of Gastroenterology, University of Milano-Bicocca and Consultant Hepatologist, the Niguarda Liver Transplant Centre, Milan. “PBC is a progressive liver disease that can lead to liver failure and in some people the need for liver transplantation. So, this new medicine, that has demonstrated its potential in managing disease progression, as well as reducing itch, a symptom that can have a detrimental effect on patients’ quality of life, is positive news for doctors and their patients.”

“PBC is a very personal disease which affects everyone differently. Some people may be more symptomatic with extreme fatigue or severe itch. For others there are no symptoms, but their biomarker levels are poor, suggesting the disease is not being controlled. This means that an individualized approach to management and treatment is needed for each patient,” said Patient Advocate, Mrs Sindee Weinbaum from European Liver Patients’ Association. “Patients need to be able to partner with their doctors and have constructive conversations about what they personally need to manage the PBC they are living with. So, it is great news to have a new treatment choice for the many people who are living with PBC that is not under control.”