PLX-200 Drug Granted Orphan Drug Designation To Treat Krabbe Disease Therapy

Release Date: 04-Oct-2021

PLX-200 Drug granted Orphan Drug Designation from FDA for the treatment of Krabbe Disease Therapy. The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals. The drug PLX-200 works by binding to the retinoid X receptor-andalpha;, which binds to peroxisome proliferator-activated receptor alpha (PPARandalpha;). Activation of PPARandalpha; boosts lysosome biogenesis through transcription factor EB up regulation. The investigational drug has also been shown to reduce inflammation and prevent apoptosis.


Krabbe disease is a rare inherited disorder caused by mutations in the GALC gene that lead to a deficiency of the lysosomal enzyme, galactocerebrosidase. This leads to the accumulation of galactosylsphingosine in the central and peripheral nervous systems causing demyelination and death in children within the first two years of life.

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