Polaryx Therapeutics, Inc. (“Polaryx”), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation for PLX-200 to treat Krabbe disease.
Krabbe disease is a rare, genetic disorder caused by the deficiency of lysosomal enzyme, galactocerebrosidase (GALC). When GALC is dysfunctional or reduced in the amount, galactosylsphingosine accumulates in the central and peripheral nervous systems, resulting in demyelination and leading to death in affected children within the first two years of life.
Under the U.S. Orphan Drug Act, the FDA’s Office of Orphan Products Development provides sponsors with special status and incentives to facilitate the drug development for rare disease affecting fewer than 200,000 people in the U.S. Orphan Drug Designation provides seven years of market exclusivity if the drug candidate receives regulatory approval together with tax credits for qualified clinical trial cost, exemptions from certain FDA application fees, and assistance in clinical trial design.
“Granting by the FDA of Orphan Drug Designation for PLX-200 in Krabbe disease supports the use of PLX-200 to treat key lysosomal storage disorders with unmet medical needs. 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. We are moving forward with the necessary development steps to move into the clinical study as soon as possible,” says Dr. Hahn-Jun Lee, M.Sc., Ph.D., President and CEO of Polaryx Therapeutics, Inc.
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