Polaryx Therapeutics Receives FDA Orphan Drug Designation for PLX-200 to Treat Krabbe Disease

 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.

Alex Yang, J.D., LLM, President and CEO of Mstone Partners Hong Kong and Chair of the Board at Polaryx Therapeutics, stated that "With this additional designation, we have now comprehensively covered all of our target indications to be successfully designated as orphan drugs. We are very excited to swiftly move towards clinical trials and commercialization of our several drug candidates in a number of these highly unmet lysosomal storage disorders."

Polaryx  Therapeutics, Inc.

Polaryx Therapeutics, Inc. is developing drug candidates for lysosomal storage disorders, for which there are currently no safe and patient-friendly treatment options available. Lysosomal storage disorders are a group of rare inherited genetic disorders caused by the dysfunction or absence of lysosomal enzymes and/or molecules important in the function of these enzymes. Young children with these devastating and lethal diseases suffer due to the lack of effective treatment options. 


PLX-200 is a repurposed drug that binds to the retinoid X receptor-α (RXRα), which binds to PPARα. PLX-200 also activates PPARα, which enhances 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).

Krabbe Disease

Krabbe disease (KD; globoid cell leukodystrophy) is a rare, inherited metabolic disorder that affects approximately 1 in 100,000 individuals in the United States. KD is caused by the deficiency of lysosomal enzyme, galactocerebrosidase (GALC). The function of GALC is to catabolize the cytotoxic lipid, galactosylsphingosine (also known as psychosine). When the level of GALC or its activity is compromised, galactosylsphingosine accumulates in the central and peripheral nervous systems, ultimately leading to a neurodegenerative, demyelinating phenotype in KD. As a result, KD typically leads to death within the first two years of life.

Media Contact

Hahn-Jun Lee, M.Sc., Ph.D.



Source: Polaryx Therapeutics, Inc.

About Polaryx Therapeutics, Inc

Polaryx Therapeutics, Inc. is solely dedicated to developing drug candidates for late infantile neuronal
ceroid lipofuscinosis (LINCL) and other forms of NCL, for which there is currently no patient-friendly
treatment option available.

Polaryx Therapeutics, Inc
140 E. Ridgewood Avenue, Suite 415, South Tower,
Paramus, NJ

More Press Releases