#Sweden #FDA #Stockholm #Type 1 Diabetes #Diamyd Medical

Diamyd Medical Moves Forward with FDA for Accelerated Approval of Diamyd®

Diamyd Medical has recently reported significant advancements regarding the accelerated approval of its innovative treatment, Diamyd® (rhGAD65/alum), aimed at addressing Stage 3 Type 1 Diabetes. Following a constructive Type C meeting with the U.S. Food and Drug Administration (FDA) in December 2024, the company has received the final minutes from this meeting, which confirm several critical alignments necessary for progressing to the accelerated approval pathway.

Key Focus of the FDA Meeting

The meeting, which yielded positive outcomes, predominantly focused on refining study protocols and the analysis strategies for Diamyd®. These enhancements are crucial as they set the groundwork for an expedited review process. The discussions fell under several key areas:

Accelerated Approval Pathway Confirmation

First and foremost, the FDA confirmed that the ongoing DIAGNODE-3 Phase 3 trial aligns with its requirements for gaining an accelerated approval status. This trial aims to leverage interim efficacy data focusing on stimulated C-peptide levels as a primary endpoint. Approximately 170 evaluable participants are anticipated to complete their 15-month assessments by early 2026, providing insightful data supporting the efficacy of Diamyd®.

Safety Dataset and Confirmatory Evidence

Another reassuring aspect was the concurrence on the safety dataset and the confirmatory evidence deemed suitable for potential accelerated approval. The safety dataset, which will be assessed at the time of filing, includes data accumulated from DIAGNODE-3, along with insights from other intralymphatic trials with Diamyd® and previous placebo-controlled studies cited in the original meta-analysis published by Hannelius et al. in Diabetologia (2020).

Co-Primary Endpoints Agreement

The agreement established on co-primary endpoints is also significant. The simultaneous evaluation of stimulated C-peptide levels and HbA1c at the 24-month final analysis will help track the drug's effectiveness holistically. This comprehensive evaluation provides a well-rounded insight into the drug's performance and ensures solid evidence is available for both accelerated and full approvals.

Statistical Analysis Plan Acceptance

Furthermore, the plan for statistical analysis put forth during the meeting was accepted by the FDA, ensuring meticulous data integrity. Such rigorous standards are expected to facilitate both accelerated and comprehensive approval processes, and the FDA looks favorably on the testing strategies provided.

Insights from Recent Meta-Analysis

Another key highlight of the meeting was the FDA's emphasis on the insights derived from the Trial Outcome Markers Initiative (TOMI) meta-analysis published in Lancet (2023). This meta-analysis emphasizes the critical association between C-peptide preservation and subsequent clinical outcomes in patients with Type 1 Diabetes. The FDA agrees that the need for preserving C-peptide levels to bolster an argument for accelerated approval can be further deliberated in the upcoming pre-BLA meeting.

Orphan Drug Designation

In addition, the FDA reaffirmed the Orphan Drug Designation for Diamyd® for treating a specific subset of Type 1 Diabetes patients who exhibit residual beta cell function. This designation signifies the therapy's potential to meet notable unmet medical needs within this patient group.

About Diamyd Medical

Founded with the mission to advance precision medicine, Diamyd Medical is focused on developing therapies that can effectively prevent and treat Type 1 Diabetes and Latent Autoimmune Diabetes in Adults (LADA). Diamyd® is a groundbreaking immunomodulatory therapeutic that plays a crucial role in preserving endogenous insulin production. The drug boasts several prestigious designations, including Orphan Drug Designation and Breakthrough Designation, awarded by the U.S. FDA.

The ongoing efforts include patient recruitment for the DIAGNODE-3 Phase III trial at 60 clinics across eight European countries and the United States. Previous studies have shown strong results, and the company maintains a commitment to optimizing the treatment response via innovative methods, such as administering Diamyd® through superficial lymph node injections. This technique aims to improve results meaningfully, ensuring rapid medical breakthroughs.

As the company continues to innovate within the healthcare landscape, a biomanufacturing facility is also being developed in Umeå, Sweden, to produce the primary component, recombinant GAD65 protein, needed for Diamyd® therapy. Diamyd Medical is also proud to hold significant shares in NextCell Pharma AB and MainlyAI AB, aligning with its vision for future healthcare advancements. Diamyd Medical's B shares are traded on the Nasdaq First North Growth Market under the ticker DMYD B.

With the FDA meeting confirming numerous pivotal aspects, including the acceptance of statistical analysis plans and safety datasets, the path towards accelerated approval for Diamyd® appears set for favorable momentum, bringing hope to those affected by Type 1 Diabetes.