EMA issues qualification opinion for islet autoantibodies as enrichment biomarkers for T1D prevention trials

Critical Path Institute's (C-Path) Type 1 Diabetes Consortium (T1DC) today announced that the European Medicines Agency (EMA) has issued a positive qualification opinion for pancreatic islet autoantibodies as enrichment biomarkers for type 1 diabetes (T1D) prevention trials. The purpose of this model-based qualification is to make publicly available tools to assist in the identification and selection of patients with a likelihood of progressing to a T1D clinical diagnosis, in trials of reasonable duration.

This regulatory endorsement will provide sponsors with the confidence to use islet autoantibodies in the optimization of clinical trials evaluating novel therapies focused on the delay and/or prevention of T1D. The models used to generate the underlying evidence for this EMA qualification opinion utilize islet autoantibody status with other patient features to identify a patient's risk of progressing to a T1D diagnosis. Positivity for two or more of the autoantibodies, together with other patient features, will be used for enrichment of clinical trials focusing on the delay or prevention of the clinical diagnosis of T1D.

In their qualification opinion, EMA reiterated the unmet drug development need that T1DC targeted with this work, stating, "There is clearly an unmet need for biomarkers to aid development in T1DM prevention, a field with a long history of failed trials." EMA's qualification opinion statement says, "Positivity to at least 2 of the following islet AAs; IAA, GAD65, IA-2, and ZnT8 is qualified for use as enrichment biomarker, in combination with clinical parameters (sex, baseline age, blood glucose measurements from the 120-minute timepoints of oral glucose tolerance test (OGTT), and hemoglobin A1c (HbA1c) levels) in T1D prevention trials targeting individuals at risk of developing T1D."

The incidence of T1D is on the rise worldwide, particularly in children. In Europe, incidence rates are between 0.2 and 0.5%, with steep rises in the number of children and young people being diagnosed in certain European countries. The ability to identify individuals at risk of progressing to a clinical diagnosis of T1D is a valuable opportunity to enrich clinical trials testing interventions that can potentially delay and ultimately prevent T1D.

This qualification from the EMA would not have been possible without the tireless dedication and collaboration of the T1D research community. JDRF applauds EMA's decision to support and accelerate more innovation in therapies to delay and prevent T1D."

Marjana Marinac, Pharm.D., JDRF Senior Director of Regulatory Affairs and T1DC Co-Director

"This endorsement from EMA is a result of many years of extensive work and extraordinary collaboration among clinical researchers, patient advocacy groups, nonprofit organizations, and members of the biopharmaceutical industry facilitated by T1DC at C-Path," said Klaus Romero, M.D., M.S., F.C.P., C-Path Chief Scientific Officer. "This success was only possible through collaborative patient-level data sharing across stakeholders, and we are grateful to our many collaborators for their continued support. We look forward to the impact this tool will make in the development of novel therapies to treat the early stages of T1D with the goal of delaying or preventing the clinical onset of this disease."

The Qualification Opinion can be found on the EMA website, or on the T1D Consortium website.

The consortium is also working on the next regulatory milestone: the endorsement of these islet autoantibodies by the U.S. Food and Drug Administration. C-Path's T1D Consortium will achieve the regulatory endorsement of the islet autoantibodies currently used in clinical practice to diagnose T1D by employing the resources of all its members and engaging with regulatory agencies at each step of the process with funding and input from The Leona M. and Harry B. Helmsley Charitable Trust, Janssen Research & Development, LLC, JDRF International, Novo Nordisk A/S and Provention Bio.

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