Accelerated FDA Approval Granted for Treatment of Rare Autoimmune Liver Disease

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Ipsen announced today the accelerated FDA approval for Iqirvo (elafibranor) to treat primary biliary cholangitis (PBC), a rare liver disease affecting the bile ducts in the liver. The approved dose comes in the form of a daily 80mg tablet and can be prescribed immediately in the United States, according to a press release published this morning.

This approval is based on positive results from a phase 3 ELATIVE trial published in the New England Journal of Medicine last November.

Primary biliary cholangitis is a life-long, rare autoimmune disease characterized by the inflammation and eventual destruction of small bile ducts in the liver. After bile ducts are destroyed, bile builds up in the liver, which leads to liver failure. Primary biliary cholangitis currently impacts about 100,000 people in the United States and most commonly affects women.

Iqirvo can be administered as a solo therapy or in combination with ursodeoxycholic acid. It works by reducing the amount of alkaline phosphatase in the liver, which can build up to cause liver failure. The current list price is $11,500 for a month’s supply, according to Reuters.

The most common negative side effects of Iqirvo were weight gain, abdominal pain, diarrhea, nausea and vomiting, which occurred in at least 10% of study participants. Fractures also occurred in 6% of participants on Iqirvo. In addition, women of reproductive age on Iqirvo should be aware of potential contraceptive interactions and should use non-hormonal birth control options for at least three weeks after their last dose of Iqirvo.

“People living with PBC can feel like the symptoms they experience are dismissed by family members, friends or even their doctors, because they have not experienced something similarly disruptive in their lives. People with PBC may also feel uncertainty around the disease progression and if, or when, their liver health may deteriorate,” Carol Roberts, executive president of PBCers, a patient advocacy organization in the United States providing support to people living with PBC, said in a press release. “Earlier diagnosis and education about PBC, along with new treatment options are important to meet the current needs of people living with PBC.”

Iqirvo has not received approval outside of the United States. The confirmatory trial, called ELFIDENCE, is ongoing.