The U.S. Food and Drug Administration (FDA) has designated Hanmi Pharmaceutical's novel drug, efpegerglucagon, as a Breakthrough Therapy for rare diseases. Hanmi is set to release Phase 2 results in the latter half of this year.

Industry sources reported on the 5th that the FDA granted Breakthrough Therapy Designation (BTD) to Hanmi's Efpegerglucagon (HM15136), a drug developed to treat Congenital Hyperinsulinism (CHI).

BTD is a fast-track program designed to expedite approval for drugs that show substantial clinical improvement over existing treatments, as evidenced by data.

Drugs with BTD status receive comprehensive guidance and support from the FDA throughout the development process, from clinical trials to approval.

The designation allows for rolling review during the approval application, enabling partial data submission and increasing the chances of priority review.

Lee Moon Hee, Hanmi Pharmaceutical's clinical team leader, said the company will leverage the BTD to maintain close dialogue with the FDA to efficiently design and execute Phase 3 clinical trials.

Efpegerglucagon has already secured Orphan Drug Designation (ODD) from the FDA, the European Medicines Agency (EMA), and the Korean Ministry of Food and Drug Safety (MFDS) for the treatment of Congenital Hyperinsulinism. The FDA has also classified it as a Rare Pediatric Disease (RPD) treatment.

Moreover, the EMA recognized efpegerglucagon as an orphan drug for the treatment of autoimmune hyperinsulinemic syndrome, underscoring the drug's broad potential.

Congenital Hyperinsulinism is a rare condition characterized by excessive insulin production, resulting in hypoglycemia. Currently, there are no FDA-approved treatments specifically targeting Congenital Hyperinsulinism.

Existing approved treatments for managing hypoglycemia caused by hyperinsulinemia are limited in efficacy to specific genotypes and can lead to side effects like heart failure. As a result, patients often resort to off-label medications or ultimately undergo pancreatic surgery, accepting the associated risks.

Hanmi Pharmaceutical is developing efpegerglucagon as a groundbreaking once-weekly treatment for Congenital Hyperinsulinism, aiming to overcome the limitations of current therapies.

Hanmi is currently conducting global Phase 2 clinical trials for efpegerglucagon, with results expected to be announced in the second half of this year.

Choi In Young, head of Hanmi's R&D Center, said that the FDA's Breakthrough Therapy designation clearly indicates both the urgent need for the drug and the FDA's confidence in its development potential, adding that the company is committed to focusing all its resources on accelerating development to bring hope to patients suffering from the condition.

The U.S. Food and Drug Administration (FDA) has designated Hanmi Pharmaceutical's novel dr …

FDA Grants Breakthrough Therapy Designation to Hanmi's Efpegerglucagon: What This Means for Rare Disease Treatment