Provention Bio rollercoaster continues with generally positive remarks from FDA ahead of Thursday’s adcomm
Early last month, Provention Bio’s shares cratered after disclosing that the FDA found the pharmacokinetic profiles of its potential type 1 diabetes drug, acquired from Eli Lilly, differed when manufactured by Lilly versus Provention.
At the time, the FDA said its concerns meant that it was not ready to start post-marketing and label discussions with the company.
But now, two days ahead of the FDA’s Endocrinologic and Metabolic Drugs Advisory Committee meeting to discuss the drug, known as teplizumab, the FDA sounded a relatively positive tone on safety and efficacy. The company’s stock was up about 30% early Tuesday.
While noting some missing data from the company’s trials, the FDA said Tuesday that “because the amount of missing data was small, sensitivity analyses performed using different missing data handling approaches show that the observed efficacy by the primary analysis method is sufficiently robust to withstand conservative approaches in handling missing data.”
The question of what constitutes substantial evidence of effectiveness will be top of mind for the committee to address, FDA notes, explaining how Provention submitted a relatively small, single placebo-controlled trial with the primary endpoint of delay of clinical type 1 diabetes, as well as additional data, which FDA said can be used as “confirmatory evidence.”
On safety, the briefing documents did not reveal any glaring red flags, although the agency noted that “slightly more than 10% of patients” in the trial “were not able to receive the full course of teplizumab secondary to meeting protocol-specified withdrawal criteria.”
The committee on Thursday will be tasked with not only discussing the clinical meaningfulness of the observed median 2-year delay of onset of type 1 diabetes demonstrated in the company’s main study, but voting on an up or down approval question ahead of the FDA’s decision, which is due by July 2.
