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PBC Treatment Landscape Evolves with New Drug Approvals and Regulatory Scrutiny

• The FDA approved Iqirvo (elafibranor, Ipsen/Genfit) as a second-line treatment for primary biliary cholangitis (PBC) in patients with inadequate response or intolerance to UDCA. • Livdelzi (seladelpar, Gilead) also received accelerated FDA approval for PBC, offering another option for patients who do not respond to or cannot tolerate UDCA. • An FDA advisory committee voted against the full approval of Ocaliva (obeticholic acid), raising concerns about its long-term safety and efficacy data. • These developments mark a significant shift in the PBC treatment paradigm, introducing new therapeutic options and challenging the existing market landscape.

The primary biliary cholangitis (PBC) treatment landscape is undergoing a significant transformation with the recent FDA approvals of Iqirvo (elafibranor, Ipsen/Genfit) and Livdelzi (seladelpar, Gilead), alongside increased regulatory scrutiny of Ocaliva (obeticholic acid, Intercept Pharmaceuticals). These changes address the unmet needs of patients who do not adequately respond to or cannot tolerate ursodeoxycholic acid (UDCA), the current first-line treatment.

New Therapeutic Options for PBC

Iqirvo (elafibranor), a first-in-class oral, once-daily peroxisome proliferator-activated receptor agonist, received accelerated approval for use in combination with UDCA for adults with an inadequate response to UDCA, or as a monotherapy for those intolerant to UDCA. This approval was based on encouraging data that led to its breakthrough therapy designation in 2019.
Livdelzi (seladelpar), a selective peroxisome proliferator-activated receptor delta agonist, also gained accelerated FDA approval for PBC treatment. It is indicated for use with UDCA in adults who do not respond adequately to UDCA or as a monotherapy for those who cannot tolerate UDCA.

Ocaliva's Uncertain Future

In contrast to these approvals, an FDA advisory committee voted against the approval of obeticholic acid (Ocaliva) for PBC without cirrhosis or compensated cirrhosis with portal hypertension. The committee cited concerns about the drug's long-term safety and efficacy data, based on findings from postmarketing requirement confirmatory trial 747-302 and observational study 747-405. The committee voted 13 to 1 with no abstentions that the benefits of obeticholic acid on clinical outcomes in patients with PBC could not be verified with available data, raising “concern for real possible harm”.

Expert Perspectives on the Evolving Landscape

Experts in hepatology emphasize the importance of effective treatment strategies to improve patient outcomes and quality of life in PBC. The introduction of Iqirvo and Livdelzi represents a significant change, offering new options for patients who have not found adequate relief with existing therapies. However, the future role of Ocaliva in the treatment paradigm remains uncertain following the FDA advisory committee's unfavorable vote.
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Reference News

[1]
Drug rivals face off in PBC after FDA hands down 2 new approvals, 1 advisory snub - Healio
healio.com · Oct 10, 2024

The PBC drug market sees two new FDA-approved therapies, Iqirvo and Livdelzi, alongside an FDA advisory committee's unfa...

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