Rezolute Receives Breakthrough Therapy Designation from FDA for Ersodetug to Treat Tumour-Induced Hypoglycaemia
Tuesday, May 06, 2025
Rezolute, Inc., a late-stage company focused on rare diseases, has received Breakthrough Therapy Designation (BTD) from the US Food and Drug Administration (FDA) for its investigational treatment, ersodetug. This designation is for the treatment of hypoglycaemia caused by tumour-related hyperinsulinism (HI).
The decision was based on data from clinical studies and real-world evidence collected through the company’s Expanded Access Programme. Patients in the United States with tumour HI have already been treated with ersodetug, showing promising results. BTD is granted to treatments for serious or life-threatening conditions when early clinical evidence suggests significant improvement over current options.
Rezolute is preparing to begin a registrational clinical study for ersodetug in patients with tumour HI by mid-2025. Initial results from this trial are expected in the second half of 2026. Alongside this, the company will continue discussions with the FDA to finalise the data requirements needed for a Biologics License Application (BLA) to support approval for the tumour HI indication, building on its current programme for congenital HI.
Earlier in 2025, ersodetug had already received BTD for treating hypoglycaemia caused by congenital HI. This condition is being studied separately in an ongoing Phase 3 trial.
Tumour HI is a rare disorder caused by either islet cell tumours (ICTs) or non-islet cell tumours (NICTs). Both tumour types lead to hypoglycaemia by overstimulating insulin receptors. Insulinomas, the most common form of ICTs, trigger excess insulin production, while certain NICTs—such as hepatocellular carcinoma—produce insulin-like substances such as IGF-2 that also activate insulin receptors. Due to the high risk and limited treatment options, there is a strong need for new therapies targeting hypoglycaemia in these patients.
Ersodetug is a fully human monoclonal antibody that works by binding to the insulin receptor and reducing over-activation caused by insulin or IGF-2. It is designed to act independently of the pancreas, which makes it potentially suitable for treating all forms of HI, whether congenital or acquired.
Source: globenewswire.com
