faced a setback after a US Food and Drug Administration鈥檚 (FDA) Advisory Committee (AdCom) meeting on the prospective use of its drug Ocaliva in pre-cirrhotic fibrosis due to nonalcoholic steatohepatitis (NASH) did not go in its favour.
Twelve of the 16 AdCom members gave out a negative vote on whether the benefits of 25mg of Ocaliva outweigh the risks in NASH patients with Stage 2 or Stage 3 fibrosis available data. While the efficacy data looked promising, the risk-benefit ratio and the challenges to mitigating the risk are too substantial and it is difficult to put this into context without clinical data, said Christopher S. Coffey, PhD, professor at the Department of Biostatistics at the University of Iowa, who in response to this question.
While there is clear data on safety risks with Ocaliva, there is only evidence of potential efficacy on surrogate endpoint data, which means it is impossible to ensure a good risk-benefit profile, said Dr. James Floyd, associate professor of Medicine at the University of Washington, Seattle.
Two committee members decided to abstain, but two members voted yes to the question on whether the data supported Ocaliva鈥檚 risk-benefit profile. While there are concerns, the lack of options and the potential empowerment of patients with an approved treatment convinced another panel member, Dr. Steve Solda, associate professor of Clinical Medicine at the University of Pennsylvania鈥檚 Perelman School of Medicine, to vote yes.
Additionally, 15 members voted in favour of deferring approval until further clinical data from the Phase III 747-303 trial (NCT02548351) is submitted by the company, which could then be used for a traditional full approval pathway, rather than an accelerated one.
This marks another significant development in the race for approval for treating NASH where Intercept is competing with and its treatment resmetirom. A new drug application (NDA) filing for resmetirom remains on track for Q2 2023, based on a 9 May company update. The application for Ocaliva鈥檚 use in NASH has a PDUFA target action date on 22 June.
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By GlobalDataAdCom verdicts and votes are considered by the FDA in potential approvals, but the agency is not required to follow them. Still, this is not the first time that Intercept鈥檚 Ocaliva faced challenges in the field. In June 2020, Intercept received a complete response letter (CRL) from the FDA regarding the drug鈥檚 previous NDA. The drug鈥檚 Phase III REVERSE trial (NCT03439254) also did not meet its primary endpoint, based on a September 2022 announcement.
Also known as obeticholic acid, Ocaliva is marketed for use against primary biliary cholangitis. Recently, a fixed-dose combination of Ocaliva with bezafibrate received orphan drug status for use in the same indication. Ocaliva is a farnesoid X receptor agonist.
