TGA gives green light to Ipsen’s first-in-class PPAR in rare disease

Ipsen’s first-in-class PPAR is Therapeutic Goods Administration (TGA) approved for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in adults unable to tolerate UDCA.

The registration of oral Iqirvo (elafibranor) is an important milestone, as currently, therapeutic options which aim to slow disease progression for patients with PBC are limited, particularly in patients where treatment with UDCA alone is unsuccessful.

Professor Simone Strasser, Chair of the Clinical and Scientific Committee at the Liver Foundation, said this announcement will be welcome news to healthcare professionals and patients.

“As not all patients respond to first-line treatments, having additional treatment options is crucial for effective health management. The registration of a novel therapy is promising news for both 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” Professor Strasser said.

The approval of Iqirvo hinges on the pivotal phase III ELATIVE trial, which demonstrated a statistically significant treatment benefit on cholestasis with a 47% placebo-adjusted difference (p<0.001) between patients on Iqirvo (51%) compared with patients on placebo (4%) achieving a biochemical response.

Despite being a rare disease, the prevalence of PBC in Australia is on the rise, and is particularly prevalent amongst women, who account for 95% of the patient population.

Christina Xinos, Medical Director at Ipsen in Australia, said “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.”

She added, “Ipsen is proud to support people living with PBC and we trust that the introduction of new and innovative treatments, like Iqirvo, will provide patients and healthcare professionals with renewed hope for slowing disease progression.”

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