The U.S. Food and Drug Administration (FDA) has granted accelerated approval to Ipsen’s Elafibranor (Iqirvo) for the treatment of primary biliary cholangitis (PBC), according to a press release published on Monday.
This therapy is the first medicine to be approved in nearly a decade for the treatment of PBC, the press release stated.
PBC is an autoimmune disease in which the body produces antibodies that target the bile ducts in the liver. This produces an inflammatory reaction that damages the bile ducts and leads to bile accumulation in the liver. In time, this bile accumulation (cholestasis) can harm the liver, leading to liver failure and cirrhosis.
There is currently no cure for the disease; however, drugs such as ursodeoxycholic acid (UCDA) can slow down disease progression. A significant percentage of patients don’t respond adequately to UCDA, so there is high interest in the development of new treatments and therapies for PBC.
Elafibranor is thought to reduce bile acid synthesis, thus preventing its deleterious accumulation in hepatic tissue. Results from the ELATIVE clinical trial, published in the New England Journal of Medicine, showed that patients with PBC thatreceived Elafibranor had a significant improvement in laboratory markers correlated to cholestasis Is worth noting that the majority of patients in the trial received elafibranor in conjunction with UCDA.
Adverse reactions present in over 10% of patients receiving the drug included mostly gastrointestinal symptoms such as abdominal pain, diarrhea, nausea, and vomiting, as well as muscular complaints such as muscle pain and muscle fiber rupture (rhabdomyolysis).
“Data from the pivotal Phase III ELATIVE clinical trial demonstrated that Iqirvo is an effective second-line treatment for patients with PBC with favorable benefit and risk data,” said Dr. Kris Kowdley, Director at Liver Institute Northwest, Washington and a primary investigator on the ELATIVE study. “The approval of Iqirvo will allow healthcare providers in the U.S. to address an unmet need with the potential to significantly reduce ALP levels for our patients with PBC.”
To this date, the drug has only received approval in the United States and has been evaluated by European and British authorities.
