Elafibranor

GENFIT's November 2024 corporate investor presentation covers forward-looking statements, ACLF disease state, market opportunity, and pipeline updates, including elafibranor's potential approvals and milestones.

FDA declined full approval for obeticholic acid (Ocaliva) in treating primary biliary cholangitis (PBC), citing unfavorable benefit-risk profile. Intercept Pharmaceuticals will collaborate with the FDA on next steps, while the drug remains on the market. European Commission revoked obeticholic acid's marketing authorization for PBC in September.

The FDA issued a Complete Response Letter to Intercept Pharmaceuticals’ obeticholic acid (Ocaliva) for primary biliary cholangitis (PBC), unable to approve its supplemental New Drug Application in its current form. The decision aligns with a negative opinion from a Gastrointestinal Drugs Advisory Committee meeting in September 2024, which found insufficient evidence to verify obeticholic acid’s benefits on clinical outcomes in PBC and its favorable benefit-risk profile. Despite the CRL, obeticholic acid remains available for PBC treatment under accelerated approval status in the US.

Elafibranor received FDA accelerated approval for second-line PBC treatment, followed by seladelpar. ELFIDENCE, ELSPIRE, and ELFINITY trials assess elafibranor's efficacy and safety in PBC patients with compensated cirrhosis, incomplete UDCA response, and real-world settings, respectively.

Ipsen reports YTD 2024 total sales growth of 9.2% at CER, driven by Dysport, Cabometyx, and Bylvay. Regulatory approvals for Iqirvo and Kayfanda, and increased 2024 guidance: total-sales growth >8.0% at CER, core operating margin >31.0%.

FDA delays full approval for Intercept Pharmaceuticals' Ocaliva (obeticholic acid), originally due 15 October, pending further review. The drug, inherited by Alfasigma in 2023, faces setbacks including a 2018 FDA boxed warning and a 2024 advisory committee vote against full approval. Despite European Commission withdrawal and subsequent reinstatement, Ocaliva now competes with Gilead's Livdelzi and Ipsen's Iqirvo in the PBC treatment market.

FDA extends review of Intercept Pharmaceuticals' sNDA for full approval of obeticholic acid (Ocaliva) for primary biliary cholangitis (PBC), with no new action date provided. Despite a negative advisory committee opinion on its clinical outcomes and benefit-risk profile, Ocaliva remains available under accelerated approval.

The PBC drug market sees two new FDA-approved therapies, Iqirvo and Livdelzi, alongside an FDA advisory committee's unfavorable vote on Ocaliva's long-term safety and efficacy.

Johnson & Johnson proposed changes to the 340B drug discount program, but backed down after HRSA deemed them inconsistent with the statute. The 340B program, established in 1992, aims to help underserved communities by allowing eligible hospitals and clinics to purchase outpatient prescription medications at discounts. Recent regulatory developments include FDA approvals for Exact Sciences' Cologuard Plus, Partners Fresenius Kabi and Formycon's Otulfi, Eli Lilly's Retevmo, Sanofi and Regeneron's Dupixent, Bristol Myers Squibb's Cobenfy, and others. Additionally, Pfizer withdrew Oxbryta from the market due to safety concerns.

The European Commission approves Ipsen's IQIRVO (elafibranor) for primary biliary cholangitis, the first new therapy in the EU in a decade. Other companies, including CymaBay, Zydus, and GlaxoSmithKline, are preparing to enter the market. The approval is based on the Phase III ELATIVE trial, showing a 47% clinical benefit of IQIRVO combined with UDCA. Additionally, Gilead Sciences' LIVDELZI (seladelpar) received FDA accelerated approval for PBC.