EMA committee recommends Iqirvo approval for PBC in Europe
Decision on therapy, approved last month in US, expected by September
A European Medicines Agency (EMA) committee has recommended the approval of Ipsen‘s Iqirvo (elafibranor) as a second-line therapy for adults with primary biliary cholangitis (PBC).
Iqirvo is intended for use either alone in patients who can’t tolerate ursodeoxycholic acid (UDCA), the first-line PBC therapy, or in combination with UDCA in those who respond poorly to that treatment.
The European Commission will now review the recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP), with a decision expected by September.
The positive recommendation follows Iqirvo’s approval in the U.S. last month for that same indication.
“PBC can progress to liver damage and even liver failure without effective therapies. Today’s decision takes us closer to being able to offer Iqirvo as a new treatment for patients [in Europe],” Christelle Huguet, PhD, Ipsen executive vice president and head of research and development, said in a company press release.
According to Huguet, Iqirvo “significantly improves biomarkers that predict disease progression, without worsening symptoms.”
Recommendation for Iqirvo approval based on ELATIVE trial data
PBC is a rare liver disease marked by chronic inflammation of the bile ducts. This tube network carries bile out of the liver, where it is produced, to the intestines, where it helps break down fatty molecules during digestion.
Chronic inflammation in patients leads to cholestasis, or slowed bile flow, that over time can make bile accumulate inside the liver. That causes damage and tissue scarring, and allows bile to leak into the bloodstream, leading to symptoms such as itchy skin.
Iqirvo, originally developed by Genfit, modulates the activity of the PPARs proteins, which are known to regulate the activity of genes involved in PBC-related processes, including inflammation and scarring.
CHMP’s recommendation was based on top-line data from an ongoing Phase 3 clinical trial, called ELATIVE (NCT04526665), showing that the study met its primary goal.
Specifically, results showed that Iqirvo was better than a placebo at reducing levels of liver damage markers, namely alkaline phosphatase (ALP) and total bilirubin, after one year, in adults who failed to respond or couldn’t tolerate UDCA.
Additional findings also demonstrated the therapy’s superiority over the placebo at improving itch-related quality of life, in terms of both itch severity and its impact on sleep and emotional well-being.
Committee also favors approval of Ipsen’s odevixibat for Alagille syndrome
In a simultaneous move, CHMP also recommended Ipsen’s odevixibat for approval in Alagille syndrome, a rare genetic disease that causes body-wide problems, including cholestasis in early infancy. The committee favored the treatment’s approval to ease itch in adults and children as young as 6 months old.
A decision on odevixibat by the European Commission is also due by September. If approved, the therapy will be sold in the European Union as Kayfanda.
“With the positive opinion for Kayfanda we are moving forward in our efforts to provide a new treatment option for children with Alagille syndrome, whose liver health can deteriorate rapidly and who often endure a very poor quality of life,” Huguet said.
We are delighted to have received CHMP positive opinions for two potential new medicines in rare cholestatic liver diseases, on the same day.
Odevixibat, under the brand name Bylvay, was approved in the U.S. in October 2023 to reduce itch in Alagille patients, ages 12 months or older.
“We are delighted to have received CHMP positive opinions for two potential new medicines in rare cholestatic liver diseases, on the same day,” Huguet said, calling it “a rare achievement, and one that demonstrates our commitment to addressing the unmet medical needs in these diseases.”
About the Author
