The FDA has turned down Merck’s application to expand the indication for its cholesterol-lowering drug ezetimibe (Zetia and Vytorin, Merck). This means there’s still no end in sight for this drug’s very long and very winding road.
Ezetimibe is now indicated to reduce LDL cholesterol in people with hyperlipidemia. The proposed new indication was for the reduction of cardiovascular risk in patients with coronary heart disease and was based on the results of the IMPROVE-IT trial.
The FDA decision is not unexpected since an FDA advisory panel in December voted 10-5 against the expanded indication. The panel was in broad agreement that the treatment benefit in the trial, though it achieved statistical significance, was too small to support the label upgrade. Panel member Thomas Fleming (University of Washington) said that “the goal of clinical research should not be to achieve statistical significance.” Another panel member, Milton Packer (Baylor University), said that “the benefit here is small. It is not robust. You blink and you miss it.”
Merck did not disclose details of the FDA’s complete response letter. The company only said that it is “reviewing the letter and will determine next steps.” The commercial importance of the decision is unclear. The first generic versions of ezetimibe will be launched in the US in December of this year. In Europe the patent for Zetia expires in October 2017 and Vytorin in April 2019.