Hanmi Pharmaceutical's bio novel drug under development for a rare disease has received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA).
Hanmi Pharmaceutical said on Feb. 5 that the FDA designated the congenital hyperinsulinism (CHI) treatment "efeglucagon (HM15136)" as a breakthrough therapy (Breakthrough Therapy Designation, BTD).
Breakthrough therapy designation is a system that supports the rapid development and approval of a new drug whose potential for clinical improvement over existing therapies has been confirmed in the treatment of serious diseases. Along with intensive FDA advice, sequential review (Rolling Review), which allows partial submission of materials at the time of the approval application for review, is also permitted. The possibility of review acceleration, such as priority review, also increases.
Hanmi Pharmaceutical is developing efeglucagon as the world's first once-weekly formulation to overcome the limitations of existing treatments. Congenital hyperinsulinism is a rare disease that causes hypoglycemia due to excessive insulin secretion, and there is currently no FDA-approved treatment for this indication.
In the interim analysis of the global phase 2 trial released by the company last year, efeglucagon showed excellent safety and tolerability and significantly reduced the occurrence of hypoglycemia and severe hypoglycemia. The global phase 2 trial is proceeding smoothly, and the final results are scheduled to be released in the second half of this year.
Lee Mun-hee, GM clinical team leader (executive director) at Hanmi Pharmaceutical, said, "With the BTD designation as a starting point, we will efficiently design the phase 3 trial through consultations with the FDA and actively leverage institutional advantages such as rolling review."
Choi In-young, head of the R&D Center at Hanmi Pharmaceutical, said, "The FDA's breakthrough therapy designation is an objective indicator that reflects not only the urgent need for commercialization of the drug, but also the FDA's high assessment of its actual development potential," adding, "We will concentrate all of the company's capabilities to deliver great hope to patients suffering from the disease through faster development."
Efeglucagon had previously been designated an orphan drug (ODD) in the United States, Europe and Korea, and a rare pediatric disease (RPD) drug by the U.S. FDA.