Diamyd Medical is advancing towards an accelerated approval pathway in the United States for its antigen-specific immunotherapy, Diamyd® (rhGAD65/alum), targeting the preservation of endogenous insulin production in individuals with Stage 3 Type 1 Diabetes. The company plans to leverage an earlier interim study readout from the ongoing Phase 3 trial, DIAGNODE-3, as the foundation for a Biologics License Application (BLA) under the FDA's accelerated approval program. The interim readout is anticipated around March 2026.
Ulf Hannelius, CEO of Diamyd Medical, stated, "We are committed to working at full speed with our partners and in close dialogue with the FDA to seize this unprecedented opportunity of making Diamyd® available to patients as quickly as possible." He further emphasized the significance of the earlier readout from the DIAGNODE-3 trial and expressed confidence in Diamyd®'s disease-modifying potential, reinforced by the positive outcome of a recent futility analysis.
Regulatory Milestones and Trial Design
Diamyd Medical recently secured Fast Track designation in the U.S. for Diamyd®. The FDA has indicated that accelerated approval could be pursued based on demonstrating significant treatment-related benefits on C-peptide levels, a recognized measure of endogenous insulin production, following Diamyd® administration. The FDA's Accelerated Approval Program is designed to expedite the approval of drugs for serious conditions that address unmet medical needs, utilizing surrogate endpoints to facilitate quicker access to potentially beneficial therapies.
To support the BLA submission under the Accelerated Approval Program, Diamyd Medical intends to conduct an interim study readout encompassing efficacy data from approximately 170 participants who have completed their 15-month assessment, along with safety data from additional DIAGNODE-3 trial participants. The primary endpoint for this analysis will be C-peptide levels, a surrogate endpoint deemed reasonably likely to predict clinical benefit by the FDA. Following the interim readout, the DIAGNODE-3 trial will continue to monitor all enrolled patients until the 24-month end-of-study assessment. As of now, 148 patients have been randomized in DIAGNODE-3. The results of the interim study readout are expected around March 2026, with the precise timing contingent on the final number of participants included in the readout, followed by the submission of a BLA under the accelerated approval pathway.
DIAGNODE-3 Trial Details
The DIAGNODE-3 trial, spanning eight European countries and the United States, aims to enroll approximately 330 patients by the end of 2025, with all patients expected to complete treatment before the interim readout. The trial focuses on individuals recently diagnosed with Stage 3 Type 1 Diabetes who carry the HLA DR3-DQ2 genotype, representing approximately 40% of Stage 3 Type 1 Diabetes patients. Previous trials have indicated that this subgroup exhibits a favorable response to Diamyd®.
About Diamyd Medical
Diamyd Medical is focused on developing precision medicine therapies for the prevention and treatment of Type 1 Diabetes and Latent Autoimmune Diabetes in Adults (LADA). Diamyd® is an antigen-specific immunomodulatory therapeutic designed to preserve endogenous insulin production. It has received Orphan Drug Designation in the U.S. and Fast Track Designation from the FDA for the treatment of Stage 1, 2, and 3 Type 1 Diabetes. The confirmatory Phase III trial, DIAGNODE-3, is actively recruiting patients with recent-onset (Stage 3) Type 1 Diabetes across 60 sites in eight European countries and the U.S.
