Veru’s drug for the treatment of high-risk patients hospitalized with COVID-19 has been denied approval by the FDA advisory committee, with eight of thirteen experts voting against it in the meeting.
Study 902 was a randomized, double-blind, placebo-controlled trial of VERU-111 9 mg capsules given orally or through a nasogastric tube once daily for up to 21 days versus a placebo for reducing the risk of death from any cause at 60 days and improving other critical care outcomes.
A clear victory for Veru and its drug VERU-111 at the meeting became less likely after the FDA released its briefing document. The agency recognized that Study 902 achieved its primary objective of all-cause mortality on the 60th day.
It did, however, find some problems with the small trial’s data, such as the high death rate in the placebo group, the possibility of unblinding events, and initial disparities in the standard of care.
The advisory board’s deliberations were driven by these unanswered questions. In the end, eight experts voted against Veru and five voted for it. However, the comments from both sides showed uncertainty, as Brigham and Women’s Hospital’s Lindsey Baden, M.D., said when he explained his vote. He claimed that he agreed with voters from both sides, and that people are trying to understand the efficacy and safety signals and data.
Though Baden voted in the affirmative, he largely agreed with the justifications offered by those in the expert community who voted against it. The case against VERU-111 was laid out by Daniel Gillen, Ph.D., of the statistics department at the University of California, Irvine.
“Given the data that we have currently, I believe we have a limited both efficacy and safety data set with a new molecule where we don’t have a full understanding of the mechanism of action,” Gillen stated as he voted no. “I don’t know if we’re ever guaranteed to know that completely, but certainly we’re far from it at this point.”
Dr. Daniel Chertow of the National Institutes of Health voted in favor despite some doubts about the data, saying that he saw no obvious advantages or disadvantages. According to Chertow, the drug’s benefits outweigh its risks when considering the patient population as a whole, and since the FDA required the experts to take into account both the “known and potential” risks and benefits, this led to a positive vote from his end.
Given the uncovered medical need in COVID-19 patients who have been failed by the standard of care, Veru believes that the benefits of VERU-111 may be enough to win over the FDA and grant the company’s request for an emergency use authorization.
Veru’s application for approval was based on data from a study of over 200 patients, despite the regulator’s warning that this sample size was insufficient in comparison to other approved drugs – the typical recommendation is a 500-patient sample size. At first, the study planned to enroll 300 people hospitalized with severe COVID-19; however, this number was reduced to 90.