The Critical Path Institute (C-Path) today announced that its Type 1 Diabetes (T1D) Consortium has received a letter of support from the European Medicines Agency (EMA) to facilitate the development and validation of the proposed regulatory qualification of pancreatic islet autoantibodies commonly used in clinical practice to diagnose T1D: insulin autoantibodies, glutamic acid decarboxylase 65, and insulinoma antigen-2 autoantibodies as enrichment biomarkers for T1D clinical trials.
In their response to the T1D Consortium Letter of Intent (LOI) and Briefing Package, the EMA stated, "[Therapies that preserve endogenous β-cell function and can prevent, halt or slow T1D disease progression in a clinically meaningful way would constitute a significant advancement in T1D care.
If successful, the quantitative tools proposed by this Consortium have the potential to facilitate the streamlined design, execution, and review of clinical trials targeting this goal.]."
By the year 2050, the number of people diagnosed with T1D in the U.S. is projected to quadruple from an estimated 1.6 million in 2020 to 5 million in 2050.
The ability to screen for subjects with early stages of T1D prior to the appearance of clinical symptoms is a valuable opportunity to potentially delay, and ultimately prevent, symptomatic T1D.
The islet autoantibodies provide a means to identify patients at risk of progressing to a clinical diagnosis of T1D.
C-Path's T1D Consortium will achieve the regulatory qualification 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 he Leona M. and Harry B. Helmsley Charitable Trust, Janssen Research & Development, LLC, JDRF International, Novo Nordisk A/S, and Provention Bio.
The Leona M. and Harry B. Helmsley Charitable Trust, Janssen Research & Development, LLC, JDRF International, Novo Nordisk A/S, and Provention Bio.
JDRF is pleased the T1DC received a letter of support from EMA. This is an example of collaboration between regulators and researchers, in the public and private sectors, working together to accelerate delivery of therapies into the hands of patients."
Jessica Dunne, Ph.D., Senior Director, JDRF, and Co-Director, T1DC
This model-based qualification, will provide a tool endorsed by both the EMA and U.S. Food and Drug Administration (FDA) that utilizes islet autoantibody status, along with other relevant patient features, to identify and select patients with a likelihood of progressing to a T1D clinical diagnosis.
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 prevent T1D.
"We are delighted that the EMA is strongly supporting the development of quantitative tools that can accelerate drug development in T1D," said C-Path's Executive Director of the T1D Consortium Inish O'Doherty, PhD. "
This work has been enabled through the collaboration of the T1D community and the sharing of patient level data. We're excited to move forward with this important project which will help pave the way for forthcoming therapies with the ability to treat early stages of T1D and delay or prevent the clinical, currently irreversible, stage of the disease."
The consortium is currently working on the next regulatory milestones: the execution of the modeling analysis plan to inform the full Briefing Package for submission to the EMA and development of a Qualification Plan for submission to the FDA.