US FDA Grants Orphan Drug Designation for GS-100 Drug

Release Date: 04-Sep-2021

Grace Science receives orphan drug designation from FDA for GS-100, an investigational AAV9 gene replacement therapy to treat NGLY1 deficiency. NGLY1 deficiency is an ultra-rare genetic disease caused by loss of function mutations in the NGLY1 gene. NGLY1 initially affects children and manifests as a complex neurological syndrome, includes cognitive impairment, movement disorders, global developmental delay, abnormal tear production (alacrima) etc. Currently, there is no approved therapy for NGLY1 deficiency.


The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S and it also gives several benefits, like tax credits for qualified clinical trials, potential seven years of market exclusivity after approval and exemption from user fees.

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