Hanmi Pharmaceutical Achieves FDA Breakthrough Designation for Revolutionary Rare Disease Treatment
In a significant regulatory milestone that could transform treatment for one of medicine's most challenging rare diseases, South Korea-based Hanmi Pharmaceutical announced on February 5, 2026, that the FDA has granted Breakthrough Therapy Designation to efpeglucagon (HM15136) for treating congenital hyperinsulinism (CHI). This designation positions the investigational therapy as a potential game-changer for patients suffering from a condition that currently has virtually no effective treatment options.
Addressing a Critical Unmet Medical Need
Congenital hyperinsulinism represents one of the most devastating rare pediatric conditions, characterized by excessive insulin secretion that leads to severe, life-threatening hypoglycemia. The disease affects newborns and children, causing dangerously low blood sugar levels that can result in permanent brain damage, developmental delays, and even death if not properly managed.
What makes this regulatory recognition particularly significant is the stark reality of current treatment options. Diazoxide remains the only FDA-approved therapy specifically for hyperinsulinism, but it works for only a subset of patients and carries serious side effects including fluid retention, heart failure, and excessive hair growth. For patients who don't respond to diazoxide, treatment options become increasingly desperate, often requiring off-label medications or ultimately pancreatic resection surgery despite its severe complications.
"The FDA's Breakthrough Therapy Designation is an objective indicator that the FDA has given high marks not only for the urgent need for commercialization of this drug but also for its actual development potential," said Choi In-young, Head of Hanmi Pharmaceutical's R&D Center. "We will concentrate all of the company's capabilities to bring great hope to patients suffering from this disease through faster development."
Revolutionary Once-Weekly Formulation
Efpeglucagon represents a sophisticated approach to treating congenital hyperinsulinism through its novel mechanism as a long-acting glucagon analog. Unlike current treatments that attempt to reduce insulin production, efpeglucagon works by counteracting insulin's effects through sustained glucagon activity, helping maintain stable blood glucose levels.
The therapy's once-weekly formulation addresses a critical limitation of existing treatments, which often require multiple daily doses and frequent monitoring. This convenience factor could prove transformative for families managing a child with congenital hyperinsulinism, reducing the burden of constant medication administration while potentially providing more consistent glucose control.
According to interim analysis results from Hanmi's global Phase 2 clinical trial announced last year, efpeglucagon demonstrated excellent safety and tolerability while significantly reducing both hypoglycemic and severe hypoglycemic episodes. The company is currently conducting the global Phase 2 clinical trial and plans to announce complete results in the second half of 2026.
Regulatory Recognition Validates Platform Potential
The FDA's Breakthrough Therapy Designation provides Hanmi with significant regulatory advantages, including intensive consultation and support throughout development, rolling review capabilities, and expanded eligibility for accelerated approval programs. These benefits could substantially reduce development timelines and bring this potentially life-saving treatment to patients faster.
Importantly, efpeglucagon has already received multiple regulatory recognitions, including Orphan Drug Designation from the U.S. FDA, European Medicines Agency, and Korea's Ministry of Food and Drug Safety. The U.S. FDA has also designated it as a Rare Pediatric Disease treatment, while the EMA has extended orphan drug designation to include insulin autoimmune syndrome, recognizing the drug's broad therapeutic potential.
Broader Implications for Rare Disease Development
Hanmi's success in securing breakthrough designation reflects broader trends in rare disease drug development, where regulatory agencies are increasingly willing to provide expedited pathways for treatments addressing serious unmet medical needs. The designation validates the FDA's commitment to supporting innovation in areas where patients have few or no treatment options.
For the rare disease community, efpeglucagon's development represents hope that sophisticated pharmacological approaches can address conditions previously managed only through crude interventions. The therapy's potential to provide effective, convenient treatment for congenital hyperinsulinism could serve as a model for developing targeted treatments for other rare metabolic disorders.
The breakthrough designation also highlights the growing recognition of congenital hyperinsulinism as a serious medical condition requiring dedicated therapeutic development. With current treatments failing many patients and surgical interventions carrying significant risks, the medical community has long awaited more effective options.
Looking Ahead
As Hanmi advances efpeglucagon through Phase 2 trials with results expected later this year, the company is positioned to potentially deliver the first targeted treatment specifically developed for congenital hyperinsulinism. The therapy's once-weekly dosing, combined with its novel mechanism of action, could establish a new standard of care for this devastating rare disease.
For families affected by congenital hyperinsulinism, efpeglucagon represents more than just a new treatment option. It offers hope for children to live normal lives without the constant threat of severe hypoglycemia and the limitations imposed by current therapeutic approaches. As Hanmi continues its clinical development program, the rare disease community will be watching closely to see whether this innovative therapy can deliver on its promise to transform outcomes for patients with one of pediatric medicine's most challenging conditions.
