<World’s first once-weekly congenital hyperinsulinism treatment>
FDA development-acceleration measure to support expedited approval…Hanmi to announce Phase 2 results in 2H this year
(February 5, 2026) The U.S. Food and Drug Administration (FDA) has designated a rare disease biologic drug candidate being developed by Hanmi Pharmaceutical as a Breakthrough Therapy.
Hanmi Pharmaceutical announced on February 5 that the FDA on February 3 (local time) granted Breakthrough Therapy Designation (BTD) to efpegerglucagon (HM15136), a treatment under development for congenital hyperinsulinism (CHI).
The FDA’s BTD program is designed to provide various benefits to facilitate expedited approval of medicines intended to treat serious conditions, when clinical data indicate the drug may demonstrate substantial improvement over existing therapies.
A therapy designated as BTD receives intensive guidance and support from the FDA across the full development pathway―from clinical development through review and approval―and may be eligible for Rolling Review, which allows portions of a marketing application to be submitted and reviewed on a sequential basis.
In addition, eligibility for other review acceleration programs, such as Priority Review, may be expanded, which is expected to enhance the efficiency of development and regulatory review processes.
Dr. Moon Hee Lee, Head of the clinical team at Hanmi Pharmaceutical, who is leading the clinical development of efpegerglucagon, said, “Through BTD, we will continue close dialogue with the FDA to efficiently design and execute our Phase 3 clinical trial,” adding, “We also plan to make good use of the advantages of Rolling Review.”
Meanwhile, efpegerglucagon has already received Orphan Drug Designation (Orphan Drug Designation, ODD) from the U.S. FDA, the European Medicines Agency (EMA), and Korea’s Ministry of Food and Drug Safety (MFDS) as a treatment for congenital hyperinsulinism, and has also been designated by the U.S. FDA as a Rare Pediatric Disease (RPD). In addition, the EMA granted efpegerglucagon orphan designation for the treatment of insulin autoimmune syndrome, reflecting a strong recognition of the drug’s broad potential.
Congenital hyperinsulinism is a rare condition characterized by excessive insulin secretion, leading to hypoglycemia. To date, there is no FDA-approved therapy specifically indicated for congenital hyperinsulinism, and the one existing therapy approved to control hypoglycemia caused by hyperinsulinism has efficacy limited to certain genotypes and is associated with significant side effects (hypertrichosis, fluid retention, heart failure, etc.). As a result, patients are often forced to use off-label medications or ultimately rely on pancreatectomy, accepting the risk of severe side effects.
Hanmi Pharmaceutical is developing efpegerglucagon as the world’s first once-weekly treatment for congenital hyperinsulinism to overcome the limitations of current approaches.
According to interim results from a global Phase 2 clinical trial announced by Hanmi last year, efpegerglucagon demonstrated excellent safety and tolerability and showed a significant reduction in both hypoglycemia and severe hypoglycemia events. The global Phase 2 trial of efpegerglucagon is progressing smoothly, and the Phase 2 results are scheduled to be announced in the second half of this year.
In-Young Choi, Head of Hanmi’s R&D Center, said, “The FDA’s Breakthrough Therapy Designation is an objective indicator that the FDA has highly evaluated not only the urgent need to commercialize this new therapy, but also its development potential,” adding, “We will concentrate all of the company’s capabilities to deliver meaningful hope to patients suffering from this disease through faster development.”