The regulator approved the peroxisome proliferator-activated receptor agonist amid growing cases of the rare condition.
Australians with primary biliary cholangitis who aren’t benefiting from ursodeoxycholic acid (UDCA) now have access to an alternative.
The TGA recently approved elafibranor (Iqirvo, Ipsen), which is the first peroxisome proliferator-activated receptor (PPAR) agonist that regulates both PPARɑ and PPARδ.
Adults are eligible for the 80mg once daily oral medication in combination with UDCA if they have had inadequate response to UDCA alone, or as a monotherapy if they don’t tolerate UDCA.
“As not all patients respond to first-line treatments, having additional treatment options is crucial for effective health management,” Professor Simone Strasser, chair of the clinical and scientific committee at the Liver Foundation, told media.
“The registration of a novel therapy is promising news for patients affected by primary biliary cholangitis and their healthcare providers. Effective treatment of PBC can slow liver disease progression and improve outcomes including preventing the need for liver transplantation,” she said.
While the rare disease currently affects an estimated 5000 Australians, the prevalence is rising, according to a press release by manufacturer Ipsen.
The medicine can be taken with or without food, and the dose doesn’t need to be adjusted for patients with renal impairment, those over age 65 or patients with mild (Child-Pugh A) or moderate (Child-Pugh B) hepatic impairment.
Nevertheless, it is not recommended for patients with severe hepatic impairment (Child-Pugh C), or patients who have or develop decompensated cirrhosis.
Liver function and blood creatine phosphokinase (CPK) tests should be done prior to initiation and again in routine patient management.
“If increases in liver biochemical tests and/or liver dysfunction are observed, prompt investigation of the cause is recommended and interruption of elafibranor treatment should be considered,” the product information says.
“Increases in blood creatine phosphokinase (CPK) have been reported in participants receiving elafibranor (3.7% in elafibranor group compared to 0% in placebo group).
“In addition to these reported CPK increases, there was one case of rhabdomyolysis which occurred in the pivotal phase 3 ELATIVE study in a participant with cirrhosis and ongoing treatment with an HMG- CoA reductase inhibitor.”
The drug may cause harm to the fetus if taken during pregnancy, and is not recommended during pregnancy or in women of childbearing age who aren’t on effective contraception.
According to the ELATIVE study, cholestasis appeared to improve in patients taking elafibranor. A biochemical response was found in 51% of those taking the drug compared to 4% of those on the placebo.
Patients taking the medication also had a significant reduction from baseline in alkaline phosphatase within a month, sustained through to a year of treatment.
The most common adverse events that were higher in the treatment group compared with placebo were abdominal pain, diarrhoea, nausea and vomiting.
“PBC is a debilitating chronic condition that, if not treated, can result in serious health complications. The registration of Iqirvo is an important milestone, as it will provide another option for up to 40% of patients who do not respond adequately to first-line treatment and have previously had limited alternatives,” Christina Xinos, medical director of Ipsen in Australia, told media.
