Genexine Inc., an innovative biotechnology company focused on immuno-oncology, metabolic and orphan diseases, announced that US FDA Office of Orphan Products Development (OOPD) has granted GX-H9 an orphan drug designation for the treatment of growth hormone deficiency.

GX-H9 is next-generation, long-acting recombinant human growth hormone (rhGH) consisting of recombinant growth hormone genetically fused to Genexine’s proprietary hybrid Fc (hyFc) platform, giving it extended half-life and potentially better safety profile. Compared to the current daily injection therapy, GX-H9 is being evaluated as weekly and twice-monthly dosing regimens to treat Growth Hormone Deficiency (GHD).

The US FDA’s Orphan Drug designation is a program devised to advance the evaluation and expedite development of drugs and biologics which demonstrate significant promises for the diagnosis or treatment of rare diseases or life-threatening conditions affecting fewer than 200,000 people in United States. The designation provides the sponsor with development and commercial supports such as prioritized FDA review on clinical trials and approvals, credits on clinical trials, exemption from certain regulatory fees and 7 years of market exclusivity.

“We are thrilled to announce the first FDA Orphan designation of our pipeline. The Orphan Drug designation for GX-H9 will enable us to move one step faster towards our goal of providing convenience to adults and kids suffering from GHD, especially in the largest GH market of the US, stated Dr. Michael Keyoung, president and chief executive officer of Genexine.

Genexine presented its multinational phase 2 interim data from both pediatric and adult GHD trials at The European Society for Paediatric Endocrinology’s 55thAnnual Meeting 2016 (ESPE) in September and will present at The 9thBiennial Asia Pacific Pediatric Endocrinology Society/50th Annual Japanese Society for Pediatric Endocrine Society Conference in Tokyo on November 19th. Genexine will obtain full data from AGHD trial by the end of this year and interim primary endpoint data from PGHD trial in 1H, 2017.