Diamyd Medical receives positive feedback from FDA on potential Accelerated Approval for Diamyd® in Type 1 Diabetes

Diamyd Medical received encouraging feedback from the U.S. Food and Drug Administration (FDA) regarding an Accelerated Approval pathway for its immunotherapy, Diamyd^®, for treating patients with Stage 3 Type 1 Diabetes carrying the genotype HLA DR3-DQ2.

In a recent in-person Type C Meeting the FDA acknowledged that C-peptide could be used by Diamyd Medical as a surrogate endpoint reasonably likely to predict the clinical benefit of preservation of endogenous insulin production. As such, Accelerated Approval could be sought based on a demonstration of significant treatment-related benefits on C-peptide levels in response to Diamyd^® administration.  Diamyd Medical has and will continue to have ongoing discussions with the FDA to determine the requirements for a Biologics License Application (BLA) under an Accelerated Approval pathway, including a potential earlier readout of stimulated C-peptide from the ongoing Phase 3 trial DIAGNODE-3.

“Aligning with the FDA on the pathway for Accelerated Approval for Diamyd represents a significant step towards making this treatment available to patients with type 1 diabetes," says Ulf Hannelius, CEO of Diamyd Medical. "The potential of using C-peptide as a surrogate endpoint for accelerated approval marks an important milestone. We are committed to working closely with the FDA to diligently advance on this pathway and to obtain regulatory approval as soon as possible for this promising therapy.” 

Professor Mark Atkinson, PhD, Diamyd Medical Board Member and an investigator with four decades of efforts seeking attempts to prevent and reverse type 1 diabetes, noted, “I left this FDA meeting with an extreme sense of optimism that a pathway for approval is feasible for Diamyd.  The need for such a therapy clearly exists, and the real winners of any such decision, if afforded, will be those living with type 1 diabetes, and perhaps even those who have yet to be diagnosed.”

About Accelerated Approval and Surrogate Endpoints
The FDA instituted its Accelerated Approval Program to allow for earlier approval of drugs that treat serious conditions, and fill an unmet medical need based on a surrogate endpoint.  A surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign or other measure that is thought to predict clinical benefit but is not itself a measure of clinical benefit. The use of a surrogate endpoint can considerably shorten the time required prior to receiving FDA approval.

About Diamyd Medical
Diamyd Medical develops precision medicine therapies for the prevention and treatment of Type 1 Diabetes and LADA (Latent Autoimmune Diabetes in Adults). Diamyd^® is an antigen-specific immunomodulatory therapeutic for the preservation of endogenous insulin production that has been granted Orphan Drug Designation in the U.S. as well as Fast Track Designation by the U.S. FDA for the treatment of Stage 1, 2 and 3 Type 1 Diabetes. DIAGNODE-3, a confirmatory Phase III trial is actively recruiting patients with recent-onset (Stage 3) Type 1 Diabetes in eight European countries and in the US. Significant results have previously been shown in a large genetically predefined patient group - in a large-scale meta-analysis as well as in the Company’s prospective European Phase IIb trial, where Diamyd^® was administered directly into a superficial lymph node in children and young adults with recently diagnosed Type 1 Diabetes. Injections into a superficial lymphnode can be performed in minutes and are intended to optimize the treatment response. 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^®. Diamyd Medical also develops the GABA-based investigational drug Remygen^® as a component in the treatments of metabolic diseases. Diamyd Medical is a major shareholder in the stem cell company NextCell Pharma AB and in the artificial intelligence company MainlyAI AB.

Diamyd Medical’s B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B. FNCA Sweden AB is the Company’s Certified Adviser.