Rezolute, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ersodetug for the treatment of hypoglycemia due to tumor hyperinsulinism (HI). This designation aims to encourage the development of treatments for rare diseases, offering benefits such as seven years of market exclusivity and potential expedited pathways to approval.
Addressing Unmet Needs in Tumor Hyperinsulinism
Tumor HI is a rare condition characterized by hypoglycemia resulting from excessive activation of the insulin receptor, driven by either islet cell tumors (ICTs) or non-islet cell tumors (NICTs). Insulinomas, the most common ICTs, lead to hypoglycemia through the overproduction of insulin. NICTs, such as hepatocellular carcinoma, can also cause hypoglycemia by producing insulin-like substances like IGF-2, which activate the insulin receptor. The high morbidity and mortality rates associated with tumor HI underscore the urgent need for targeted therapies.
Susan Stewart, J.D., Chief Regulatory Officer of Rezolute, stated, "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. 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."
Ersodetug: A Novel Approach to Hyperinsulinism
Ersodetug is a fully human monoclonal antibody designed to bind to a unique allosteric site on insulin receptors. This mechanism counteracts the effects of insulin receptor over-activation by insulin and related substances like IGF-2, thereby improving hypoglycemia in patients with hyperinsulinism (HI). Acting downstream from the pancreas, ersodetug has the potential to be universally effective in treating hypoglycemia caused by any form of HI.
Clinical Development and Future Plans
Ersodetug has demonstrated real-world benefits in patients with insulinoma, and preclinical studies suggest it can effectively blunt IGF-2 and insulin-mediated insulin-receptor signaling. Rezolute anticipates commencing a Phase 3 registrational trial for ersodetug in patients with tumor HI in 2025. This trial will build upon the benefits observed in the Expanded Access Program, further evaluating the drug's efficacy and safety in this patient population.
Rezolute is a late-stage rare disease company focused on improving outcomes for individuals with hypoglycemia caused by hyperinsulinism (HI). Their antibody therapy, ersodetug, is designed to treat all forms of HI and has shown substantial benefit in clinical trials and real-world use for the treatment of congenital HI and tumor HI.
