On December 3, 2024 Rezolute, Inc. (Nasdaq: RZLT) ("Rezolute" or the "Company"), a late-stage biopharmaceutical company dedicated to developing transformative therapies for rare diseases with serious unmet needs, reported the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ersodetug for the treatment of hypoglycemia due to tumor HI (Press release, Rezolute, DEC 3, 2024, View Source [SID1234648764]).

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"FDA’s granting of Orphan Drug Designation is a recognition of the serious unmet need patients with tumor hyperinsulinism face and validates the potential significant benefit that ersodetug can provide," said Susan Stewart, J.D, Chief Regulatory Officer of Rezolute. "Tumor HI requires directed treatments for hypoglycemia to prevent serious adverse outcomes and improve patients’ quality of life, as well as to enable patients to continue cancer treatments. We are thrilled with FDA’s designation, which allows us to continue developing a potential universal treatment for hypoglycemia caused by all forms of HI."

The FDA’s Orphan Drug Designation is intended to encourage the development of treatments for rare diseases. Orphan Drug Designation offers benefits including seven years of market exclusivity protection and may shorten the clinical development path through potential qualification for expedited pathways to approval.

About Tumor Hyperinsulinism (HI)

Tumor HI is a rare disease that may be caused by two distinct types of tumors: islet cell tumors (ICTs) and non-islet cell tumors (NICTs), both of which lead to hypoglycemia as a result of excessive activation of the insulin receptor. Insulinomas are the most common type of ICT and may cause hypoglycemia by stimulating the over production of insulin. A variety of different NICTs, particularly hepatocellular carcinoma, can cause hypoglycemia by producing and secreting insulin-like paraneoplastic substances such as IGF-2 that bind to and activate the insulin receptor. With high morbidity and mortality rates within tumor HI, there remains a significant unmet need for new therapies directed at hypoglycemia treatment. Ersodetug has shown real-world benefit in patients with insulinoma and preclinical studies have shown that ersodetug can similarly blunt IGF-2 and insulin-mediated insulin-receptor signaling.

About Ersodetug

Ersodetug is a fully human monoclonal antibody that binds to a unique allosteric site on insulin receptors to counteract the effects of insulin receptor over-activation by insulin and related substances (such as IGF-2), thereby improving hypoglycemia in the setting of hyperinsulinism (HI). Because ersodetug acts downstream from the pancreas, it has the potential to be universally effective at treating hypoglycemia due to any form of HI.