Provention Bio wins FDA panel backing for teplizumab to delay type 1 diabetes

An FDA advisory committee on Wednesday tipped in favour of recommending approval for Provention Bio's teplizumab to delay the onset of type 1 diabetes in at-risk individuals. The panel voted by a margin of 10 to 7 that information provided by Provention Bio demonstrates that the benefits of using the anti-CD3 antibody in this indication outweigh its risks. The FDA is expected to render a decision on the priority review filing by July 2.

The panel based its recommendation on the Phase II TN-10 study of 76 participants, ranging in age from eight to 45 years, who were randomly assigned to receive a single 14-day course of teplizumab or placebo. Results showed that teplizumab delayed insulin-dependent, clinical-stage disease by a median of at least two years in presymptomatic patients with Stage 2 type 1 diabetes.

The AdCom panel backing comes a few days after the FDA released briefing documents concluding that TN-10 had "successfully demonstrated" the treatment effect of teplizumab. However, agency reviewers did flag certain problems with the filing, notably regarding pharmacokinetic discrepancies between the drug product tested in TN-10 and the version Provention Bio intends to use for commercial sales – an issue the company previously warned could end up delaying FDA approval of the drug.

In a statement following the panel vote, CEO Ashleigh Palmer said individuals at-risk of developing type 1 diabetes "are waiting urgently for access to clinical advancements to address their significant medical needs. We remain committed to working closely with the FDA to hopefully secure approval of teplizumab and potentially bring the first disease-modifying therapy in [type 1 diabetes] to at-risk patients as soon as possible."