By Diagnostics World Staff
April 11, 2022 | Following many years of collaborative patient-level data sharing across stakeholder groups, the European Medicines Agency (EMA) recently endorsed pancreatic islet autoantibodies as enrichment biomarkers for type 1 diabetes (T1D) trials focused on delaying or preventing the disease. The Qualification Opinion appears on both the EMA website and that of the Critical Path Institute’s (C-Path) Type 1 Diabetes Consortium (T1DC).
The qualification is intended to provide publicly-accessible tools to help in the identification and selection of trial subjects with a likelihood of progressing to a T1D clinical diagnosis. The opinion is based on evidence that positivity for two or more of the autoantibodies, coupled with other patient features, can identify patients at risk of such progression.
In its qualification statement, the EMA references the unmet need for biomarkers to aid development in T1D prevention, “a field with a long history of failed trials.” Among the islet autoantibodies qualified for use as enrichment biomarkers are IAA, GAD65, IA-2, and ZnT8, together with four clinical parameters—sex, baseline age, blood glucose measurements from the 120-minute timepoints of oral glucose tolerance test, and hemoglobin A1c levels.
T1D is rising in prevalence globally, particularly in children, reports the T1DC in its post. In Europe, incidence rates are between 0.2% and 0.5%.
The consortium is now working toward endorsement of the islet autoantibodies by the U.S. Food and Drug Administration. Regulatory support will be achieved using the resources of all T1DC members and by engaging the agency at each step of the process.
Specifically mentioned was 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.