Handok and Genexine’s long-acting growth hormone for children, HL2356 (GX-H9), was designated as an orphan drug during development by the Ministry of Food and Drug Safety on March 4th.

This designation as an orphan drug during development provides Handok and Genexine with increased momentum in the development of HL2356 (GX-H9) and raises the likelihood of its future development as an orphan drug. Being designated as an orphan drug allows for an expedited review, shortening the approval period and exemption from bridging data requirements. Additionally, it may qualify for conditional approval after Phase 2 clinical trials, extension of the product license validity period, and market exclusivity rights, among other special cases.

Prior to this orphan drug designation during development, HL2356 (GX-H9) was designated in October 2023 by the Ministry of Food and Drug Safety for the GIFT Keum Consultative Group. GIFT stands for Global Innovative Products on Fast Track, a program that supports from the early stages to shorten the review period. Being a GIFT target provides various supports for rapid productization, including periodic concurrent reviews starting with prepared materials and one-on-one expert consulting with developers.

In addition, HL2356 (GX-H9) has been designated as an orphan drug for the treatment of growth hormone deficiency by the U.S. Food and Drug Administration (FDA) in 2016 and the European Medicines Agency (EMA) in 2021.

HL2356 (GX-H9) is a long-acting growth hormone that applies Genexine’s long-acting platform technology, hyFcTM. Handok and Genexine are jointly developing “HL2356 (GX-H9)” for children and adults with the goal of once-weekly administration. Growth hormones are widely used not only for diseases such as growth delay and developmental disorders due to growth hormone deficiency in children, but also for hormone therapy for the prevention of adult diseases and anti-aging. “Growth hormone deficiency” is a rare and intractable disease in which growth hormone secretion is deficient due to causes such as pituitary damage, genetic defects, or tumors in the pituitary gland or hypothalamus.

HL2356 (GX-H9) secured significant results that met the primary endpoints in a Phase 3 clinical trial for children in China last year. I-Mab Biopharma, which holds the rights in China, plans to apply for new drug approval in China this year based on these results.