Diamyd Medical is moving closer to an accelerated approval pathway for its antigen-specific immunotherapy, Diamyd® (rhGAD65/alum), for Stage 3 Type 1 Diabetes, following a positive Type C meeting with the U.S. Food and Drug Administration (FDA) in December 2024. The meeting focused on refining the study protocol and analysis strategies for Diamyd®. The FDA has confirmed that the ongoing DIAGNODE-3 Phase 3 trial aligns with requirements for an Accelerated Approval, leveraging interim efficacy data based on stimulated C-peptide as the primary endpoint. Approximately 170 evaluable participants are expected to complete their 15-month assessments by early 2026.
FDA Feedback on Development Program
The FDA provided affirmative feedback on several pivotal aspects of Diamyd Medical's development program. This includes concurrence on the suitability of both the safety dataset and the confirmatory evidence for a potential accelerated approval. The safety dataset will include data from DIAGNODE-3 at filing, along with data from other intralymphatic trials with Diamyd® and the three placebo-controlled trials evaluating subcutaneous injections of Diamyd® from the meta-analysis published by Hannelius et al. in Diabetologia (2020). The FDA also agreed that DIAGNODE-3 serves as the pivotal trial to demonstrate substantial evidence of effectiveness, supported by DIAGNODE-2 and the aforementioned placebo-controlled trials as confirmatory evidence.
Endpoints and Statistical Analysis
Agreement was reached on the simultaneous evaluation of stimulated C-peptide levels and HbA1c as co-primary endpoints at the 24-month final analysis. The FDA has accepted the testing strategy for endpoint evaluation, ensuring robust data integrity for both accelerated and full approvals.
The FDA emphasized that the Trial Outcome Markers Initiative (TOMI) meta-analysis, published by Taylor et al. in Lancet (2023), provides critical insights into the association between C-peptide preservation and clinical outcomes in type 1 diabetes. The FDA agreed that the justification for the required level of C-peptide preservation to support an accelerated approval, including its relationship to a clinically meaningful reduction in HbA1c, could be further discussed at a pre-BLA meeting and included in the Biologics License Application (BLA).
Orphan Drug Designation
Additionally, the FDA reaffirmed the Orphan Drug Designation for Diamyd® for treating a subset of Type 1 Diabetes patients with residual beta cell function, highlighting the therapy's potential to address significant unmet needs in this well-defined patient population.
Breakthrough T1D Support
Diamyd Medical will receive USD 1.75 million in expanded collaboration support from Breakthrough T1D (formerly JDRF) in support of the DIAGNODE-3 Phase 3 trial to facilitate the potential submission of a regulatory filing for approval. This additional funding builds upon the existing collaboration to support the ongoing DIAGNODE-3 trial evaluating a precision medicine antigen-specific immunotherapy for autoimmune diabetes.
Ongoing Clinical Trial
The early readout, planned for March 2026 for the ongoing Phase 3 DIAGNODE-3 trial, will include approximately 170 evaluable participants who have completed their 15-month assessment. As of today, 191 patients have been randomized in the trial. This analysis will provide efficacy data based on preservation of C-peptide levels, which the FDA recognizes as a surrogate endpoint reasonably likely to predict clinical benefit in Type 1 Diabetes, and can therefore be used to obtain Accelerated approval.
About Diamyd Medical
Diamyd Medical develops precision medicine therapies to prevent and treat Type 1 Diabetes and LADA (Latent Autoimmune Diabetes in Adults). Diamyd® is an antigen-specific immunomodulatory therapeutic for the preservation of endogenous insulin production. Diamyd® has been granted Orphan Drug Designation in the U.S. as well as Breakthrough Designation and Fast Track Designation by the U.S. FDA for the treatment of Stage 3 Type 1 Diabetes. DIAGNODE-3, a confirmatory Phase III trial is actively recruiting patients with recent-onset (Stage 3) Type 1 Diabetes at 60 clinics in eight European countries and in the US. A biomanufacturing facility is under development in Umeå, Sweden, for the manufacture of recombinant GAD65 protein, the active ingredient in the antigen-specific immunotherapy Diamyd®.
