Diamyd Medical's Major Progress Towards FDA Accelerated Approval for Diamyd® Therapy

Recent FDA Meeting Advances Diamyd Medical's Therapeutics

Diamyd Medical has made significant headway in its efforts to secure accelerated approval for its immunotherapy product, Diamyd® (rhGAD65/alum), designed specifically for treating Stage 3 Type 1 Diabetes. The pivotal developments stem from a recent Type C meeting with the U.S. Food and Drug Administration (FDA) held in December 2024, during which key protocols and analysis strategies where discussed and refined.

In a move that brings promise to the Type 1 Diabetes community, the FDA provided final meeting minutes documenting a positive outcome and confirming alignment on critical developmental milestones. These milestones include the acceptance of the ongoing DIAGNODE-3 trial as pivotal for achieving accelerated approval status based on interim efficacy data, particularly focusing on stimulated C-peptide as the primary endpoint from approximately 170 evaluable participants. These participants are set to complete their assessments by the start of 2026.

Moreover, the FDA affirmed the robustness of DIAGNODE-3’s safety dataset, which is integral for securing accelerated approval. This dataset not only comprises results from the ongoing trial but also includes evidence from historical intralymphatic trials involving Diamyd®, alongside three placebo-controlled studies documented in a major meta-analysis published in Diabetologia.

Co-Primary Endpoints and Future Plans

In light of the meeting, both the FDA and Diamyd Medical have reached an agreement on co-primary endpoints, which will include evaluations of stimulated C-peptide levels and HbA1c at the 24-month mark of the final analysis. Acceptance of the statistical analysis plan was also a key focus, ensuring a solid foundation for data integrity in forthcoming evaluations that seek to facilitate both accelerated and full approvals.

The FDA underscored the importance of the analysis presented in the Trial Outcome Markers Initiative (TOMI) meta-analysis, emphasizing its role in elucidating the relationship between C-peptide preservation and clinical outcomes in type 1 diabetes. The dialogue surrounding C-peptide preservation's impact on achieving a clinically relevant decrease in HbA1c will continue, with future discussions planned for a pre-BLA meeting where this crucial aspect will be further elaborated.

Additionally, the FDA has retained the Orphan Drug Designation for Diamyd®, further solidifying its commitment to addressing the significant unmet needs of patients who maintain some residual beta cell function, a well-defined subgroup within the broader spectrum of Type 1 Diabetes.

About Diamyd Medical

Diamyd Medical is at the forefront of developing precision medicine targeting Type 1 Diabetes and Latent Autoimmune Diabetes in Adults (LADA). Their innovative treatment, Diamyd®, is engineered specifically to preserve the body’s own insulin production—a critical aspect of managing these notoriously difficult conditions. In recognition of its potential, Diamyd® has not only secured Orphan Drug Designation in the U.S. but has also been awarded Breakthrough Designation and Fast Track Designation by the FDA for Stage 3 Type 1 Diabetes, alongside similar track designations for earlier stage Type 1 scenarios.

The company is actively involved in the DIAGNODE-3 Phase III trial across 60 clinics in eight European nations and the U.S., recruiting patients who have been newly diagnosed with Type 1 Diabetes. Past studies have already demonstrated promising outcomes in genetically predetermined patient cohorts, including findings from both a large-scale meta-analysis and a European Phase IIb trial where the treatment was successfully administered via injection directly into a lymph node for optimal response.

As part of their commitment to innovation, a biomanufacturing facility is being established in Umeå, Sweden, to ensure a steady supply of recombinant GAD65 protein, the active ingredient in their immunotherapy. Additionally, Diamyd Medical holds significant stakes in NextCell Pharma AB, focused on stem cell advancements, and MainlyAI AB, which leverages artificial intelligence.

As the company moves forward, the anticipation surrounding the future of Diamyd® remains high, especially with its listing on the Nasdaq First North Growth Market, under the ticker DMYD B.