The U.S. Food and Drug Administration has granted accelerated approval to Iqirvo (elafibranor) for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA.
PBC Background
PBC is a rare, chronic, progressive liver disease in which the bile ducts become inflamed and damaged. This leads to the buildup of bile and causes liver problems such as scarring, cirrhosis, and eventual liver failure. Many people do not have symptoms when they are first diagnosed and may not develop symptoms for several years. Early symptoms may include fatigue, itchy skin, and abdominal pain. As the disease progresses, people with PBC may develop:
- Weakness
- Nausea
- Diarrhea
- Swelling in the legs and feet
- bBone and joint pain
- Jaundice
- Dark urine
- Xanthomas
PBC is considered an autoimmune disease in which the immune system malfunctions and mistakenly attacks a person’s healthy bile duct cells, causing the inflammation and damage. It is thought to be caused by a combination of genetic susceptibility and environmental triggers.
Elafibranor
Elafibranor is an oral, once-daily, first-in-class of medication called peroxisome proliferator-activated receptor (PPAR) agonists. This is the first treatment approved for PBC in some time. In 2016, the FDA approved obeticholic acid in combination with UDCA for adults who have an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA.
The accelerated approval follows data from the Phase 3 ELATIVE study, where it was observed that 13 times more patients achieved the primary endpoint of biochemical response when treated with elafibranor versus placebo plus UDCA. Secondary endpoints also showed normalization in alkaline phosphatase levels (ALP), a biochemical marker used as an endpoint in PBC trials. Data from this trial was recently published in the New England Journal of Medicine.
To learn more about PBC and other rare gastrointestinal diseases, visit https://checkrare.com/diseases/gastrointestinal-diseases/