Immunotherapy to Delay, Treat Type 1 Diabetes Wins FDA Fast Track Designation

The US FDA granted Fast Track designation for Diamyd (rhGAD65/alum), an antigen-specific immunomodulatory therapeutic that preserves endogenous insulin production. The novel drug treats type 1 diabetes (T1D) in children with stage 1 or stage 2 T1D who carry the HLA DR3-DQ2 genotype, according to the July 18 announcement from Swedish company Diamyd Medical.¹

The designation is the second this year for rhGAD65/alum after Diamyd announced earlier that the drug received the Fast Track nod for treating individuals with stage 3 type 1, or frank diabetes, who carry the same genotype. The FDA also previously granted Orphan Drug status to rhGAD65/alum.¹

"We are thrilled to receive our second Fast Track designation for Diamyd, this time for its potential to delay the onset of clinically diagnosed type 1 diabetes", Ulf Hannelius, CEO of Diamyd Medical, said in the news release. "This recognition once again underscores...urgent need for new treatment options for type 1 diabetes across the disease spectrum. We now have the opportunity to work closely with the FDA to accelerate the development of Diamyd also as a preventive medicine."¹

A chronic autoimmune disorder, type 1 diabetes may evolve slowly, beginning months or even years before symptomatic disease occurs. The autoimmune response triggers a cascade of reactions that leads to steady destruction of pancreatic β cells. The progression of T1D occurs in 3 stages characterized by the presence of antibodies and the state of glucose homeostasis.

In stage 1, individuals have tested positive for 2 or more diabetes-specific autoantibodies, but have no symptoms, and their glucose levels remain normal. In stage 2 T1D, autoantibodies are still present, but the hallmark of disease progression is dysglycemia. Stage 2 also does not produce symptoms. In stage 3 T1D, glucose levels are dangerously erratic; stage 3 is synonymous with clinically diagnosed T1D.¹

Learn from experts about screening and staging in type 1 diabetes

Diamyd Medical is evaluating rhGAD65/alum in a series of trials under the ASSET program, funded by Sweden’s innovation agency VINNOVA.² The safety, feasibility, and immune response of the drug, which is administered via injection into a superficial lymph node, is being evaluated in individuals with stages 1 or 2 T1D in the DiaPrecise trial. Individuals with stage 3 T1D, ie, clinically diagnosed T1D, are the target population for the company’s confirmatory and pivotal DIAGNODE-3 phase III trial. DIAGNODE-3 is actively recruiting in the US and in 8 European countries with a planned enrollment of 330 individuals aged 12 to 28 years.^1,2

Diamyd has reported significant results from a large, genetically predefined population evaluated in a large-scale meta-analysis and in DIAGNODE-2, a prospective European phase 2b trial in children and young adults recently diagnosed with type 1 diabetes.^1,2

The active ingredient in the antigen-specific immunotherapy is recombinant glutamic acid decarboxylase 65 protein, a primary autoantigen in T1D. Injections into a superficial lymph node can be performed in minutes and are intended to optimize the treatment response, according to the company.^1,2

References
1. Diamyd Medical receives second US FDA Fast Track designation for Diamyd - for the prevention of type 1 diabetes. News release. Diamyd Medical. July 18, 2024. Accessed July 18, 2024. https://prnmedia.prnewswire.com/news-releases/diamyd-medical-receives-second-us-fda-fast-track-designation-for-diamyd---for-the-prevention-of-type-1-diabetes-302200410.html
2. Diamyd Medical receives first regulatory approval to start the Phase III trial DIAGNODE-3 with the diabetes vaccine Diamyd. News release. Diamyd Medical. September 21, 2021. Accessed July 18, 2024. https://www.diamyd.com/docs/pressClips.aspx?ClipID=4070572