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

The antigen-specific immunotherapy preserves endogenous insulin production via intralymphatic injection of recombinant GAD65 protein, according to Diamyd Medical.

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



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