Diamyd Medical's Diamyd® Receives Positive FDA Feedback for Accelerated Approval in Type 1 Diabetes
Key Insights
Diamyd Medical (search) received positive feedback from the FDA regarding an Accelerated Approval pathway for Diamyd® in Stage 3 Type 1 Diabetes (search) patients with the HLA DR3-DQ2 genotype.
The FDA acknowledged C-peptide as a surrogate endpoint, which could allow for Accelerated Approval based on demonstrating significant treatment-related benefits on C-peptide levels.
A Phase 3 trial, DIAGNODE-3, evaluating the safety and efficacy of Diamyd® in Type 1 Diabetes (search), had a successful interim analysis, recommending continuation without modifications.
Diamyd Medical (search) has announced positive feedback from the U.S. Food and Drug Administration (FDA) regarding a potential Accelerated Approval pathway for its immunotherapy, Diamyd®, in treating patients with Stage 3 Type 1 Diabetes (search) who carry the genotype HLA DR3-DQ2. This development marks a significant step toward making the treatment available to a specific subset of patients with type 1 diabetes, addressing a critical unmet need in preserving endogenous insulin production.
FDA Acknowledges C-Peptide as Surrogate Endpoint
During a recent Type C Meeting, the FDA acknowledged that C-peptide could be used by Diamyd Medical (search) as a surrogate endpoint reasonably likely to predict the clinical benefit of preservation of endogenous insulin production. This acknowledgment is pivotal because it opens the door for Accelerated Approval based on demonstrating significant treatment-related benefits on C-peptide levels in response to Diamyd® administration. Accelerated Approval is granted for drugs treating serious conditions that fill an unmet medical need, based on a surrogate endpoint that predicts clinical benefit but is not itself a measure of clinical benefit.
Ulf Hannelius, CEO of Diamyd Medical (search), stated, "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 (search). 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."
DIAGNODE-3 Trial Shows Positive Interim Analysis
The confirmatory Phase III trial DIAGNODE-3 (www.diagnode-3.com), evaluating the safety and efficacy of Diamyd® in individuals diagnosed with Type 1 Diabetes (search), is ongoing in the United States and in eight European countries. The trial will enroll up to 330 individuals aged 12 to 29 years, recently diagnosed (within six months) with Type 1 Diabetes, who carry the HLA DR3-DQ2 haplotype, a genetic risk factor for this disease.
Diamyd Medical (search) also announced a successful interim analysis (non-futility test) for its ongoing Phase 3 DIAGNODE-3 trial. The interim analysis, reviewed by an independent Data Safety Monitoring Board (DSMB), resulted in a favorable recommendation to continue the trial without any modifications. The interim analysis evaluated six-month data from 74 patients enrolled in the trial, assessing the likelihood of the trial achieving one of its co-primary endpoints, specifically the preservation of endogenous insulin-producing capacity measured as stimulated C-peptide. The DSMB's recommendation is based on the data observed to date, indicating that the trial is on track and has the potential to meet its objectives.
About Diamyd® and Type 1 Diabetes
Diamyd® is an antigen-specific immunomodulatory therapeutic designed to preserve endogenous insulin production. It has been granted Orphan Drug Designation in the U.S. and Fast Track Designation by the U.S. FDA for the treatment of Stage 1, 2, and 3 Type 1 Diabetes (search). Type 1 Diabetes is an autoimmune disease where the body's immune system destroys insulin-producing beta cells in the pancreas. This leads to a deficiency in insulin, a hormone crucial for regulating blood glucose levels. The disease affects millions worldwide, and there is a significant unmet need for therapies that can preserve insulin production and improve glycemic control.
The company is actively recruiting patients for the DIAGNODE-3 trial and is in ongoing discussions with the FDA to determine the requirements for a Biologics License Application (BLA) under the Accelerated Approval pathway, including a potential earlier readout of stimulated C-peptide from the trial.