FDA Awards Orphan Drug Status to Rezolute's RZ358 for Tumor-Induced Hypoglycemia Treatment

Rezolute, Inc., a biopharmaceutical firm specializing in the development of treatments for rare diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to their drug, ersodetug. This designation is for the treatment of hypoglycemia caused by tumor hyperinsulinism (HI). The designation signifies a significant acknowledgment of the unmet medical needs of patients with this condition and underscores the potential benefits that ersodetug could offer.

Susan Stewart, Chief Regulatory Officer of Rezolute, expressed the company’s enthusiasm over the FDA's decision, noting that tumor HI requires targeted treatments to manage hypoglycemia effectively. This is crucial to not only improve patients’ quality of life but also to allow them to continue their cancer treatments without interruption. The ODD by the FDA will enable Rezolute to advance ersodetug as a comprehensive therapy for hypoglycemia resulting from all forms of HI.

The FDA’s Orphan Drug Designation aims to promote the development of medications for rare diseases by offering incentives such as seven years of market exclusivity and potential expedited approval pathways.

Tumor hyperinsulinism is a rare disorder caused by either islet cell tumors (ICTs) or non-islet cell tumors (NICTs), which both lead to hypoglycemia due to excessive insulin receptor activation. Insulinomas, the most common form of ICTs, induce hypoglycemia by over-stimulating insulin production. Various NICTs, including hepatocellular carcinoma, can produce insulin-like substances, such as IGF-2, which mimic insulin’s effects, causing hypoglycemia. Given the high morbidity and mortality rates associated with tumor HI, there remains a critical need for new therapies addressing hypoglycemia.

Ersodetug, a fully human monoclonal antibody, binds to a specific site on insulin receptors. This binding counteracts the over-activation caused by insulin and similar substances like IGF-2, thus ameliorating hypoglycemia in patients with hyperinsulinism. Since its action is downstream of the pancreas, ersodetug holds the promise of being an effective treatment for hypoglycemia due to any form of HI.

Rezolute, Inc. is committed to significantly improving outcomes for patients suffering from hypoglycemia induced by hyperinsulinism. The company’s lead therapy, ersodetug, is designed to treat all forms of HI and has demonstrated considerable benefits in clinical settings and practical applications.