Regulatory milestone highlights opportunity for collaborative regulatory science and evidence frameworks to support upcoming therapies.

/PRNewswire/ -- Critical Path Institute (C-Path), through its Type 1 Diabetes Consortium, today renewed its dedication to enhancing the regulatory science and evidence structures essential for advancing the next wave of treatments across stages of type 1 diabetes (T1D). The statement follows the U.S. Food and Drug Administration's (FDA) accelerated approval last week of Tzield as the first disease modifying therapy for stage 3 type 1 diabetes, a step achieved by Sanofi, a long standing member of C Path's T1D Consortium.

C Path is broadening the evidence foundation for C peptide as a biomarker by raising the number of trials under review to 74. With ongoing FDA engagement, this larger data collection will aid continued assessment of C peptide as a drug development tool to deliver varied classes of disease modifying therapies to individuals with T1D more rapidly.

The approval together with the expanded C peptide evidence base mark notable advances for the field and underscore the value of shared frameworks to define clinically meaningful benefit, produce confirmatory evidence and aid development of future therapies using different approaches.

The consortium's key 2025 public workshop, 21st Century Trials in New Onset Type 1 Diabetes, drew more than 400 stakeholders and reviewed scientific and regulatory issues around C peptide and preserved beta cell function as indicators of disease modification and clinically meaningful benefit. The event united varied viewpoints at a pivotal time, highlighting the worth of collaborative precompetitive regulatory discussion to tackle drug development hurdles.

Although the FDA action centers on recently diagnosed stage 3 T1D, it affirms the wider chance to build regulatory grade evidence frameworks that can guide therapeutic development across all disease stages where endogenous beta cell function stays relevant. Creating consistent methods to define clinically meaningful disease modification will grow more vital as additional therapies move through the pipeline.

The June 12 announcement represents a key moment for type 1 diabetes drug development, said Simi Ahmed, Ph.D., executive director of C Path's Type 1 Diabetes Consortium. The chance now lies in jointly developing shared regulatory science methods that define and measure the clinical importance of preserved beta cell function relative to how a person feels, functions or survives. By uniting regulators, academia, patient groups and drug developers in a neutral precompetitive setting, C Path stays committed to bolstering the infrastructure required to speed therapeutic innovation across multiple stages of type 1 diabetes.

Progress in regulatory science is collaborative and iterative, said Joseph Hedrick, Ph.D., senior advisor of the T1D Consortium. The questions that shape clinically meaningful benefit and future development paths are increasingly common across sponsors and stakeholders. By gathering diverse expertise and enabling early regulatory dialogue in a neutral environment, C Path assists the field in constructing evidence frameworks that can enhance consistency, efficiency and confidence across ongoing development programs.

The FDA's accelerated approval of the first disease modifying therapy for stage 3 type 1 diabetes stands as a landmark for the field, said Jeffrey A. Bluestone, Ph.D., distinguished professor emeritus at the University of California, San Francisco. There is substantial opportunity now for researchers and sponsors to collaborate in defining clinically meaningful benefit and constructing shared regulatory frameworks needed to accelerate development of these therapies, irrespective of disease stage or therapeutic approach.

Addressing these questions will demand sustained alignment on regulatory expectations and evidence generation strategies across the full T1D drug development ecosystem. Through its neutral precompetitive model, C Path helps the field confront foundational scientific and regulatory challenges that no single organization can efficiently resolve alone.

To learn more about C Path's Type 1 Diabetes Consortium and view the full list of member organizations, visit the C Path website.

About Critical Path Institute

Critical Path Institute (C Path) is an independent, nonprofit organization established in 2005 as a public private partnership in response to the FDA's Critical Path Initiative. C Path's mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C Path's global headquarters is in Tucson, Arizona; C Path's Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c path.org.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 56 percent funded by the FDA/HHS, totaling $23,740,424, and 44 percent funded by non government source(s), totaling $18,881,611. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.

Media Contacts:

Roxan Triolo Olivas
C Path 520.954.1634

Kissy Black
C Path
615.310.1894

SOURCE Critical Path Institute