FDA Delays Full Approval Decision for Alfagigma's Ocaliva Following Negative AdComm
核心洞察
The FDA has delayed its decision on converting Ocaliva, a drug for primary biliary cholangitis (搜索), from accelerated to full approval after a negative advisory committee vote.
An FDA advisory committee voted against full approval of Ocaliva, citing that confirmatory trial data did not verify a clinical benefit.
The European Commission also revoked Ocaliva’s conditional marketing authorization in September following a negative recommendation from the EMA's Committee for Medicinal Products for Human Use.
Following a nearly unanimous advisory committee (AdComm) vote against Alfagigma (搜索)'s Ocaliva (obeticholic acid), the FDA has delayed its decision on whether to grant full approval to the drug for primary biliary cholangitis (搜索) (PBC). The original Prescription Drug User Fee Act (PDUFA) date for the supplemental New Drug Application (sNDA) was October 16.
Ocaliva remains available in the US under accelerated approval for PBC patients. Alfagigma (搜索) acquired Ocaliva as part of its $800 million buyout of Intercept Pharmaceuticals (搜索) last year.
The FDA AdComm in September recommended against full approval for the farnesoid X receptor (FXR (搜索)) agonist by a 13-1 vote, concluding that confirmatory trial data did not verify that the treatment provided a clinical benefit. The AdComm discussed data from a traditional confirmatory study designed to be randomized and placebo-controlled, and a real-world evidence trial.
In a separate vote, panelists decided 10-1 that the benefits of Ocaliva did not outweigh its risks, with three abstentions.
The European Commission also revoked Ocaliva’s conditional marketing authorization in September, which was available in Europe as a second-line treatment for PBC. The decision followed a June recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use, which reassessed the benefit-risk profile of the drug.
Ocaliva initially received accelerated approval in 2016. However, it faced challenges, including the addition of a boxed warning in 2018 after it was being incorrectly dosed as a daily treatment instead of weekly for some patients.