Polaryx Therapeutics Receives FDA Orphan Drug Designation for PLX-200 for Treatment of Krabbe Disease

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Orphan drug designation for Polaryx’s PLX-200 will facilitate research into its use treating Krabbe Disease, a rare lysosomal genetic disorder.

Polaryx Therapeutics, a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced that FDA granted orphan drug designation for PLX-200 to treat Krabbe disease on Sept. 2, 2021.

Krabbe Disease is a rare genetic disorder caused by a deficiency in a lysosomal enzyme called galactocerebrosidase (GALC). When GALC is dysfunctional or has a reduced presence in the body, it causes galactosylsphingosine to accumulate in the central and peripheral nervous systems. This results in demyelination and can lead to death in affected children within the first two years of life.

PLX-200 is a drug that binds to the retinoid X-receptor-α, which binds to PPARα. It activates PPARα, which results in enhanced production of transcription factor EB (TFEB). TFEB then binds to a gene promoter involved in lysosome biogenesis, facilitating their production.


“Because supportive care is the only available treatment option for most cases of Krabbe disease, this designation validates our scientific rationale and strongly motivates us to expedite the clinical development of PLX-200 in Krabbe disease.” said an-Jun Lee, president and CEO of Polaryx, in a company press release. “We are moving forward with the necessary development steps to move into the clinical study as soon as possible.”

Source: Polaryx