Clinical stage, specialty biopharmaceutical company Xeris Pharmaceuticals announced that it has received Orphan Drug Designation from both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for its investigational soluble glucagons as preventive treatment for severe, persistent hypoglycemia in patients with congenital hyperinsulinism (HI).
The company’s proprietary glucagons formulation provides the first soluble, room temperature stable, pump-delivered glucagon product that enables continuous infusion to prevent high insulin levels and resulting severe hypoglycemia. Xeris revealed that the European Commission (EC) has also awarded orphan status for its soluble glucagon for the treatment of HI upon recommendation of the European Medicines Agency’s Committee for Orphan Medicinal Products (EMA COMP).
Douglas R. Baum, Xeris' CEO, said, “We are pleased that both the FDA and EMA have recognized the importance of HI and designated orphan status for our soluble glucagon product for this indication. Covered by multiple U.S. Investigational New Drug applications, we are developing several soluble glucagon products for patients with diabetes, we believe our glucagon can be useful in a number of other patient populations, such as the HI population, who suffer from severe, persistent hypoglycemia.”
Julie Raskin, Executive Director of Congenital Hyperinsulinism International (CHI), said, “Historically, many patients with diffuse HI have had to undergo surgery to remove 95 to 98 percent of their pancreas to reduce insulin secretion to a level that does not result in repeated hypoglycemic episodes. …Sadly, by 15 years of age, some 95 percent of these patients have insufficient insulin production, and they become insulin-dependent for the remainder of their lives. We are very supportive of new drug products… Xeris soluble glucagon delivered via a pump is one of just a handful of new therapies to come along in 30 years."
The orphan designation makes Xeris eligible for a range of benefits, including tax credits, waiver of certain New Drug Application (NDA) fees, and a 7-year marketing exclusivity in the U.S.
Earlier this year, Xeris launched the Phase 2 clinical study of G-Pen Mini, a room-temperature stable glucagon injection intended to simplify treatment for mild-to-moderate hypoglycemia or low blood sugar.