Critical Path Institute® (C-Path) today reaffirmed its commitment, through the Type 1 Diabetes Consortium, to strengthening the regulatory science and evidence infrastructure needed to enable the next phase of multi-stage treatment innovation for type 1 diabetes (T1D). This announcement comes after the U.S. Food and Drug Administration (FDA) last week accelerated the approval of Tzield® as the first disease-modifying therapy for stage 3 type 1 diabetes, a milestone achieved by Sanofi, one of the long-standing members of C-Path’s T1D consortium.
C-Path is expanding the evidence base for C-peptide as a biomarker by increasing the number of studies under evaluation to 74. Through continued engagement with the FDA, this expanded data pool will support the continued evaluation of C-peptide as a drug development tool that will help bring multiple types of disease-modifying therapies sooner to people living with T1D.
This approval and expansion of the evidence base for C-peptide represents a major advance for the field, while reinforcing the need for a shared framework to define clinically meaningful benefits, generate confirmatory evidence, and support the development of future treatments utilizing a variety of therapeutic approaches.
Most recently, the consortium’s pivotal 2025 public workshop, 21st Century Trials in New-Onset Type 1 Diabetes, attended by more than 400 stakeholders and explored scientific and regulatory considerations for C-peptide and preservation of beta-cell function as a measure of disease modification and clinically meaningful benefit. This workshop brought together diverse perspectives at a critical moment in the evolution of the field and reinforced the value of collaborative, pre-competitive regulatory dialogue in addressing drug development challenges.
Although FDA’s action is focused on recently diagnosed stage 3 T1D, it supports a broader opportunity to develop a regulatory-grade evidence framework that can inform treatment development across all stages of disease where endogenous beta cell function remains relevant. Establishing a consistent approach to defining clinically meaningful disease improvement will become increasingly important as the number of treatments progressing through the development pipeline increases.
“The June 12 announcement marks a significant moment in type 1 diabetes drug development,” said Dr. Simi Ahmed, executive director of the C-Path Type 1 Diabetes Consortium.
The opportunity now is to collaboratively build a shared regulatory science approach that defines and quantifies the clinical relevance of conserved beta cell functions in relation to how people feel, function, and survive. C-Path continues to strengthen the infrastructure needed to accelerate treatment innovation across the multiple stages of type 1 diabetes by bringing together regulators, academia, patient groups, and drug developers in a neutral, pre-competitive environment. ”
Dr. Simi Ahmed, C-Path Executive Director
“Advances in regulatory science are collaborative and iterative,” said Dr. Joseph Hedrick, senior advisor to the T1D Consortium. “Questions that define clinically meaningful benefits and future development paths are increasingly shared among sponsors and stakeholders. By bringing together diverse expertise and facilitating early regulatory dialogue in a neutral environment, C-Path is helping the field build an evidence framework that can improve consistency, efficiency, and reliability across ongoing development programs.”
“The FDA’s accelerated approval of the first disease-modifying therapy for stage 3 type 1 diabetes is a milestone for the field,” said Dr. Jeffrey A. Bluestone, Distinguished Professor Emeritus at the University of California, San Francisco. “There is now a tremendous opportunity for researchers and sponsors to work together to define clinically meaningful benefits and build the common regulatory framework needed to accelerate the development of these treatments, regardless of disease stage or treatment approach.”
Addressing these questions requires continued collaboration on regulatory expectations and evidence generation strategies across the T1D drug development ecosystem. Through its neutral, pre-competitive model, C-Path helps the field address fundamental scientific and regulatory challenges that no single organization can effectively address alone.
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Critical Path Research Institute (C-Path)
