Handok-Genexine, Long-Acting Growth Hormone Designated as Orphan Drug in Europe

[Asia Economy Reporter Seo So-jeong] Handok and Genexine announced on the 26th that their jointly developed long-acting growth hormone 'GX-H9' was designated as an orphan drug for the treatment of growth hormone deficiency by the European Medicines Agency (EMA) on the 19th of this month.

Growth hormone deficiency is a rare intractable disease caused by pituitary damage, genetic defects, tumors of the pituitary or hypothalamus, and other factors leading to a deficiency in growth hormone secretion. In adults, symptoms such as decreased muscle mass, increased cholesterol, and reduced bone density may appear, while in children, it can severely affect quality of life through dwarfism, growth retardation, and delayed sexual maturation.

Growth hormone is a protein drug used not only for diseases such as growth delay and developmental disorders in children due to growth hormone deficiency but also for hormone therapy aimed at preventing adult diseases and aging.

Handok and Genexine are developing the next-generation growth hormone GX-H9 for both children and adults by applying antibody fusion technology. Unlike existing growth hormones that require daily administration, GX-H9 can be administered once a week or once every two weeks.

The orphan drug designation by the European Medicines Agency is a system that provides benefits to developers to expedite research and development of treatments for intractable or life-threatening diseases.

With this orphan drug designation, GX-H9 will receive benefits such as reduced regulatory fees, support for clinical protocols, research grants, and 10 years of market exclusivity upon obtaining orphan drug approval.

Handok and Genexine have been jointly developing GX-H9 as strategic partners since 2012. The project was selected for support by the Ministry of Science and ICT's New Drug Development Project Group and received orphan drug designation from the U.S. Food and Drug Administration (FDA) in November 2016.

Currently, the two companies are preparing for multinational Phase 3 clinical trials of GX-H9 in the U.S. and Europe, while their Chinese partner I-Mab is conducting Phase 3 clinical trials in China.