Diamyd Medical's Type 1 Diabetes Immunotherapy Receives Global Non-Proprietary Name "Retogatein"
核心洞察
Diamyd Medical (搜索)'s investigational antigen-specific immunotherapy for type 1 diabetes (搜索) has been assigned the global non-proprietary name "retogatein (搜索)" by the WHO's International Nonproprietary Names Programme and the United States Adopted Names Council (搜索).
Retogatein (搜索) is designed to preserve endogenous insulin production in individuals with type 1 diabetes (搜索) who carry the HLA DR3-DQ2 (搜索) genotype, which constitutes approximately 40% of patients with type 1 diabetes in Europe and the US.
The therapy is currently being evaluated in the pivotal Phase 3 DIAGNODE-3 trial and has received Orphan Drug and Fast Track designations from the FDA.
Diamyd Medical (搜索) announced that its investigational antigen-specific immunotherapy for type 1 diabetes (搜索) has been assigned the global non-proprietary name "retogatein (搜索)" by the World Health Organization's International Nonproprietary Names (INN) Programme and the United States Adopted Names Council (搜索) (USAN). The therapy, previously referred to as Diamyd, represents a precision medicine approach targeting a specific genetic subset of type 1 diabetes patients.
Precision Medicine Approach for Type 1 Diabetes
Retogatein (搜索) (recombinant human GAD65 (搜索); rhGAD65) formulated with alum is designed to preserve endogenous insulin production in individuals with type 1 diabetes (搜索) who carry a defined HLA genotype. The therapy works by inducing antigen-specific immune tolerance to GAD65, aiming to modulate the autoimmune response responsible for beta-cell destruction and potentially slow or halt disease progression.
"The assignment of the non-proprietary name retogatein (搜索) represents an important milestone in the development of our precision immunotherapy for type 1 diabetes (搜索)," said Ulf Hannelius, CEO of Diamyd Medical (搜索). "The INN and USAN provide a standardized global identifier for the active substance, recombinant human GAD65 (搜索), which is a cornerstone of our efforts to address the unmet needs of individuals living with type 1 diabetes."
Current Clinical Development Status
The therapy is currently being evaluated in the pivotal Phase 3 trial, DIAGNODE-3, in individuals with newly diagnosed Stage 3 type 1 diabetes (搜索). The trial is actively recruiting patients with recent-onset type 1 diabetes at 57 clinics across eight European countries and in the United States. Notably, the DIAGNODE-3 trial recruits only patients who carry the HLA DR3-DQ2 (搜索) genotype, which constitutes approximately 40% of patients with type 1 diabetes in Europe and the US.
Retogatein (搜索) has received significant regulatory recognition, including Orphan Drug Designation and Fast Track Designation from the FDA for the treatment of Stage 3 (clinically diagnosed symptomatic) type 1 diabetes (搜索). Fast Track Designation has also been granted for the treatment of Stage 1 and 2 (pre-symptomatic) type 1 diabetes, with the Phase 3 trial having potential for an accelerated approval pathway in the US.
Previous Clinical Evidence
Significant results in preserving endogenous insulin production have previously been demonstrated in a large genetically predefined patient group, both in a large-scale meta-analysis and in the company's prospective European Phase 2b trial. These findings support the precision medicine approach of targeting patients with the specific HLA DR3-DQ2 (搜索) genotype.
Manufacturing and Corporate Structure
Diamyd Medical (搜索) is developing a biomanufacturing facility in Umeå, Sweden, for the manufacture of recombinant GAD65 (搜索) protein, the active ingredient retogatein (搜索) in the antigen-specific immunotherapy. The company is also a major shareholder in stem cell company NextCell Pharma AB and artificial intelligence company MainlyAI AB (搜索).
The INN/USAN designation ensures consistent identification and communication in scientific, clinical, and regulatory settings worldwide. Hannelius noted that the company will use the non-proprietary name retogatein (搜索) in scientific, clinical, and corporate communications while advancing the process toward a future trade name as they move closer to commercialization.