Ipspen and Genfit’s elafibranor has received priority review designation from the US Food and Drug Administration (FDA) for the treatment of the rare chronic liver disease PBC.

The designation means the FDA will review the candidate within six months, instead of the usual ten. The agency has set a Prescription Drug User Fee Act (PDUFA) date of 10 June 2024, according to a 7 December press release by Ipsen.

The candidate – which received FDA breakthrough therapy designation in 2019 – is designed as a second-line treatment option for patients with PBC – a rare, progressive liver disease in which the bile ducts are gradually destroyed. Currently, PBC is treated mainly with ursodeoxycholic acid (UDCA).

If approved, Ipsen and Genfit’s elafibranor will compete with Intercept’s Ocaliva (obeticholic acid). The FDA approved the drug as a second-line therapy for PBC in 2016. Earlier this year, Intercept said it would place more focus on Ocaliva’s use for PBC after it received a negative Advisory Committee (AdCom) decision for the drug’s blockbuster use in nonalcoholic steatohepatitis (NASH).

Ipsen and Genfit are seeking simultaneous regulatory submissions for elafibranor. In addition to the FDA accepting the new drug application (NDA), Ipsen said the European Medicines Agency (EMA) has validated a marketing authorisation application (MAA) for the drug. A review by the Committee for Medicinal Products for Human Use (CHMP) began on 26 October 2023. The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has also approved the candidate for review.

In December 2021, the two companies entered an exclusive licensing agreement where Ipsen gained the rights to develop and commercialise Genfit’s elafibranor. The deal involved an upfront payment of €120m ($129m) and up to €360m ($388m) in milestone payments. Genfit will also receive tiered double-digit royalties of up to 20%.

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By GlobalData

Elafibranor is an oral dual peroxisome-activated receptor alpha/delta agonist. The drug, taken once daily, targets cholestasis, bile toxicity, inflammation, and bile acid output. Ipsen and Genfit released positive results from a Phase III trial investigating elafibranor in combination with UDCA in PBC patients with intolerance or inadequate response to UDCA. The open-label, randomised, placebo-controlled ELATIVE trial (NCT04526665) met its primary composite endpoint.

Ipsen’s EVP and R&D head Christelle Huguet said: “Elafibranor, if approved, has the potential to change the management of this challenging condition for people living with PBC, offering a new second-line treatment choice, where the number of effective options is currently limited.”