Ipsen's IQIRVO Approval Boosts Primary Biliary Cholangitis Market

The European Commission has granted conditional approval for Ipsen's IQIRVO (elafibranor) for the treatment of primary biliary cholangitis (PBC) in adults, marking the first new therapy for this condition in the EU in almost a decade. This approval has spurred other companies like CymaBay Therapeutics, Zydus Therapeutics, and GlaxoSmithKline to bring their candidates into the PBC market.

Primary biliary cholangitis, previously known as primary biliary cirrhosis, is a chronic liver disease characterized by the slow destruction of bile ducts in the liver. This obstruction can lead to cirrhosis and portal hypertension over time. According to DelveInsight, there were approximately 298,000 diagnosed cases of PBC in the 7MM (US, EU5, and Japan) in 2023, with the United States accounting for nearly 48% of these cases. The condition predominantly affects women.

The primary treatment goals for PBC include slowing disease progression and managing symptoms such as itching, osteoporosis, and dry eyes/mouth. Ursodeoxycholic acid (UDCA) is the primary medication used to slow disease progression. While UDCA shows clinical, biochemical, and histological improvements in early-stage patients, its efficacy in advanced stages is uncertain. Corticosteroids are also being studied for their potential benefits, though they come with concerns like osteoporosis.

For severe cases, liver transplantation remains the only life-saving option, especially for patients with severe itching. The prognosis for PBC patients post-transplant is generally better than for other chronic liver diseases, with 5-year survival rates of 84-87% and 10-year survival rates of 79-84%.

In May 2016, the US FDA granted accelerated approval for OCALIVA (obeticholic acid) for PBC treatment. It is used in combination with UDCA for patients who do not respond adequately to UDCA or as a standalone therapy for those who cannot tolerate it. OCALIVA remains the only approved second-line therapy for PBC. The European Commission also granted conditional approval for OCALIVA later in 2016 for similar indications.

Recently, the European Commission has given conditional approval for IQIRVO (elafibranor) 80mg tablets for PBC treatment. It can be used alongside UDCA or as a standalone treatment for those intolerant to UDCA. This approval follows a positive recommendation from the EMA's Committee for Medicinal Products for Human Use and the US FDA's Accelerated Approval. Elafibranor showed a 47% clinical benefit in the Phase III ELATIVE trial, which evaluated its efficacy in combination with UDCA compared to a placebo.

In August 2024, Gilead Sciences announced that the FDA granted accelerated approval for LIVDELZI (seladelpar) for treating PBC. Seladelpar, a potent PPARδ agonist, is designed for patients who do not respond to or cannot tolerate UDCA. Gilead has also completed its acquisition of CymaBay and validated a Marketing Authorization Application for seladelpar with the EMA.

Emerging therapies such as Saroglitazar Magnesium (Zydus Therapeutics), Linerixibat (GlaxoSmithKline), and Setanaxib (Calliditas Therapeutics AB) are expected to impact the PBC market positively. Saroglitazar, which has orphan drug and fast track designations from the FDA, is in Phase II/III trials. Linerixibat, aimed at reducing itching in PBC patients, is in Phase III trials and has an orphan drug designation.

DelveInsight forecasts significant growth in the primary biliary cholangitis market, driven by advancements in therapeutic approaches, including first-line agents like UDCA and second-line treatments like obeticholic acid. The market was valued at USD 1.1 billion in the 7MM in 2023 and is expected to grow as new therapies receive regulatory approval.