MHRA approves Ipsen’s Elafibranor for PBC

Ipsen has received approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for its medication Elafibranor (Iqirvo), intended for the treatment of primary biliary cholangitis (PBC) in adults. This medication is specifically designed to enhance liver function by reducing bile acid production and decreasing inflammation in the liver. The recommended dosage of Elafibranor is one tablet per day, which should be taken consistently at the same time each day. It can be used either alone or in combination with ursodeoxycholic acid (UDCA).

The MHRA’s endorsement of Elafibranor comes after a rigorous placebo-controlled study that included 161 adult participants suffering from PBC. Most of these participants had been using UDCA for over a year and continued to do so during the trial, while some had to stop due to adverse effects. The effectiveness of Elafibranor was measured by the reduction of blood levels of alkaline phosphatase (ALP) and bilirubin, both of which are indicators of liver damage. A successful outcome was defined as normalization of these levels and at least a 15% reduction in ALP after one year of treatment.

The results of the study were promising. 51% of the participants who received Elafibranor experienced the necessary decrease in ALP and bilirubin levels. In contrast, only 4% of those who were given a placebo showed similar improvements. These findings highlight the potential of Elafibranor to significantly benefit patients with PBC.

Patients taking Elafibranor are advised to consult their healthcare providers if they experience any side effects, and to report such incidents via the MHRA’s Yellow Card scheme. Primary biliary cholangitis is a chronic liver disease that results in damage to the bile ducts within the liver, leading to bile buildup and potentially to liver failure if left untreated.

Julian Beach, the interim executive director for healthcare quality and access at MHRA, emphasized the importance of ensuring safe access to effective medications. He stated, “Enabling safe access to high quality, safe and effective medicines is a key priority for us. We’re assured that the appropriate regulatory standards of safety, quality, and effectiveness for the approval of this new formulation have been met. As with all products, we will keep its safety under close review.”

The approval of Elafibranor represents a significant advancement in the treatment options available for adults suffering from primary biliary cholangitis, offering hope for improved management of this progressive liver disease.