The long-acting recombinant human growth hormone GX-H9 being jointly developed by Handok and Genexine was designated an orphan drug for the treatment of growth hormone deficiency by the European Medicines Agency (EMA) on July 19, 2021.

Growth hormone deficiency is an intractable and rare disease in which the secretion of growth hormone is deficient due to pituitary disorders, genetic defect, or a tumor of the pituitary or hypothalamus. Adults may experience a decrease in muscle mass, an increase in cholesterol, and a decrease in bone density, while children may find their quality of life severely affected due to dwarfism, decreased growth, delayed sexual maturity, and other symptoms.

The recombinant human growth hormone (rhGH) is a protein drug in wide use not only for growth delay and developmental disorders in children due to growth hormone deficiency, but also in prevention of adult diseases and as an anti-aging hormone therapy. Handok and Genexine are applying antibody fusion technology to develop GX-H9, a next-generation growth hormone for children and adults. GX-H9 is a long-acting rhGH that is administered once a week or once every two weeks, while existing growth hormone needs to be administered daily.

EMA’s orphan drug designation grants a variety of benefits to companies developing treatments making research and development of treatments for incurable or life-threatening diseases smoother. With this designation, GX-H9 can benefit from lower regulatory fees, clinical protocol support, research subsidies, and market exclusivity for ten years upon acquisition of an orphan drug license.