To the primary biliary cholangitis (PBC) community – it’s time to rejoice! For the first time in nearly one decade, the U.S. Food and Drug administration (FDA) has approved a novel treatment for this indication. Global biopharmaceutical company Ipsen shared on June 10, 2024 that its therapy Iqirvo (elafibranor), administered in 80mg tablets, was approved under the FDA’s Accelerated Approval program.
In-licensed from GENFIT three years ago, Iqirvo is a first-in-class PPAR agonist that is taken once each day. The treatment is approved for adults with PBC who do not respond adequately to ursodeoxycholic acid or who cannot tolerate ursodeoxycholic acid. This is especially important as existing treatments like ursodeoxycholic acid often leave individuals with unmanaged symptoms and struggling with disease progression; as PBC progresses, the incidence and risk of liver failure increases significantly. Through offering better disease control, treatments like Iqirvo reduce the burden of PBC on both patients and the healthcare system.
Behind the Accelerated Approval
The accelerated approval follows available data from the Phase 3 ELATIVE clinical study. 161 participants enrolled. Of these, 108 individuals received both Iqirvo and ursodeoxycholic acid, while the remaining 53 individuals received a placebo plus ursodeoxycholic acid. Findings from the study underscore that:
- Iqirvo significantly improved biochemical response. 13x more individuals met biochemical response when receiving Iqirvo alongside ursodeoxycholic acid than those receiving just ursodeoxycholic acid alone. This means that Iqirvo lowered alkaline phosphatase (ALP) levels. Higher ALP levels correlate with cholestasis, or impaired bile flow. Lower ALP levels suggest improvements in liver function. ALP levels also normalized in 15% of treated patients within a 52-week period. In comparison, ALP levels did not normalize in any participants within the ursodeoxycholic acid monotherapy cohort.
- Irqivo was relatively safe and well-tolerated. Some side effects did occur, including abdominal pain, weight gain, nausea and vomiting, and diarrhea. This treatment may also cause muscle pain, rhabdomyolysis, hypersensitivity, drug-induced liver injury, and an increased risk of fractures.
About Primary Biliary Cholangitis (PBC)
Formerly known as: Primary biliary cirrhosis
Primary biliary cholangitis is a chronic liver disease that damages small bile ducts which carry bile out of the liver. When these ducts become increasingly damaged and inflamed, bile flow is interrupted. Bile builds up in the liver, causing liver scarring and cirrhosis. This can eventually cause liver failure. PBC predominantly affects women between ages 35-70, though it can affect both men and women of all ages. Primary biliary cholangitis is not related to alcohol consumption.
Early stages of primary biliary cholangitis are often asymptomatic. Many people do not have symptoms at the time of diagnosis, and PBC is often discovered through blood tests performed for other reasons. In these early stages, if someone is symptomatic, they may experience severe fatigue, darkened skin, small white bumps under the skin around the eyes, muscle and joint pain, dry mouth, and persistent itchiness that is worst on the palms and soles. As the disease progresses, additional symptoms may appear such as:
- Jaundice (when your skin, eyes, and mucous membranes become yellow)
- Swelling in the lower extremities
- Chronic diarrhea
- Increased risk of bone fracturing
- Internal bleeding
- Thyroid underactivity
- Abdominal ascites
