FDA Grants Breakthrough Therapy Designation to Rezolute’s Ersodetug for Hypoglycemia
Rezolute has announced Breakthrough Therapy Designation from the U.S. Food and Drug Administration for ersodetug. The designation applies to the treatment of hypoglycaemia caused by tumor-induced hyperinsulinism (HI).
The FDA’s decision is based on clinical data from across the company's development programme, alongside real-world evidence gathered through an Expanded Access Programme in the United States. BTD is designed to accelerate the development and review process for treatments targeting serious or life-threatening conditions, particularly when early clinical results suggest a significant improvement over available therapies.
Tumor-related HI presents serious challenges due to persistent and severe hypoglycaemia. Existing treatment options are often limited, especially when hypoglycaemia cannot be controlled or is a barrier to surgery or other tumor-specific interventions. The BTD recognises the therapeutic potential of ersodetug in managing this difficult-to-treat condition.
Tumor hyperinsulinism is a rare disorder caused by either islet cell tumors (ICTs), such as insulinomas, or non-islet cell tumours (NICTs), such as hepatocellular carcinoma. These tumors can lead to dangerously low blood sugar levels by overstimulating the insulin receptor. In some cases, NICTs produce substances like IGF-2 that mimic insulin and activate the same pathway, further complicating management. The condition is associated with significant morbidity and mortality, and there is a critical need for more effective therapies.
Ersodetug is a fully human monoclonal antibody that binds to the insulin receptor in a way that reduces excessive activation caused by insulin or insulin-like substances. This mechanism allows it to address both congenital and acquired forms of HI by controlling hypoglycaemia at the receptor level, rather than through the pancreas. Preclinical and real-world results have shown its potential in patients with both insulinomas and IGF-2-related hypoglycaemia.
