FDA Advisory Committee Votes In Favor Of Approval Of Provention Bio's Diabetes Med

The FDA’s Endocrinologic and Metabolic Drugs Advisory Committee voted 10-7 in favor of recommending approval of Provention Bio Inc’s PRVB teplizumab to delay the development of type 1 diabetes in high-risk children and adults, with most committee members expressing some concerns about trial size and safety.

What Happened: In briefing documents released before the meeting, the FDA noted that the study, TN-10, was “relatively small,” with only 44 patients exposed to teplizumab; however, more than 750 patients with new-onset type 1 diabetes were exposed to teplizumab in other controlled clinical studies included in a meta-analysis.

“The low prevalence of stage 2 type 1 diabetes makes it challenging to identify ‘at-risk’ patients for clinical studies, and studies of relatively long duration are needed to observe outcomes and quantify treatment benefits,” the FDA stated. 

“Furthermore, there are no approved therapies to delay the onset of type 1 diabetes, indicating an unmet need in this therapeutic area. Thus, given the severity and rarity of the disease, the lack of treatments to delay its onset, and the impracticality of conducting a second long-term clinical trial, it seems appropriate to consider this single trial plus confirmatory evidence as potentially meeting the standard for substantial evidence of effectiveness.”

See also: Best Health Insurance for Diabetics

Multiple panelists pointed to a higher rate of serious side effects in the 44 patients who received teplizumab than the 32 who got a placebo (16% versus 3%) and three patient deaths in the teplizumab arm, compared to none in the placebo group.

Related content: Benzinga's Full FDA Calendar

Why It Matters: All seemed to agree that if approved, long-term safety data will be necessary. If the drug is approved, the label should be restricted to the population enrolled in the pivotal trial.

The FDA granted Breakthrough Therapy designation to teplizumab and priority review designation for the marketing application.

The Prescription Drug User Fee Act (PDUFA) action date is July 2.

See the FDA briefing document for the meeting here.

Price Action: PRVB shares are down 8.33% at $9.79 in the premarket session on the last check Friday.