FDA Grants Orphan Drug Designation For PLX-200 Drug To Treat GM2 Gangliosidoses

Release Date: 01-Oct-2021

PLX-200 drug to treat GM2 gangliosidoses has been granted by the U.S. Food and Drug Administration (FDA). This disease is also known as Tay-Sachs and Sandhoff diseases that are ultra-rare and fatal pediatric neurodegenerative disorders. It is caused by mutations in Hexosaminidase A (HEXA) and Hexosaminidase B (HEXB), key enzymes in the lysosome, respectively.


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.


PLX-200 is a repurposed drug that binds to the retinoid X receptor-andalpha; (RXRandalpha;), which binds to PPARandalpha;. PLX-200 also activates PPARandalpha;, which improves production of transcription factor EB (TFEB). TFEB then binds to the promoter of genes involved in lysosome biogenesis and activates their production. PLX-200 also has additional activities, such as reducing inflammation and preventing cell death (apoptosis).

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