USFDA grants orphan drug designation for Polaryx Therapeutics PLX-200
Paramus: Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, has recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.
GM2 Gangliosidoses, also known as Tay-Sachs and Sandhoff diseases, are ultra-rare and fatal pediatric neurodegenerative disorders caused by defects in Hexosaminidase A (HEXA) and Hexosaminidase B (HEXB), key enzymes in the lysosome, respectively. These genetic defects lead to abnormal accumulation of gangliosides, resulting in severe progressive neurodegeneration, seizures, loss of mobility, hearing, and vision, and early death. There is no cure for these diseases and the only treatment is supportive care.
Under the U.S. Orphan Drug Act, the FDA's Office of Orphan Products Development provides sponsors with special status and incentives to facilitate 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.
"We are very pleased to be granted Orphan Drug Designation for PLX-200 from the FDA for the treatment of GM2 gangliosidoses. This designation is one of the important development milestones for the company, because it allows an expanded use of PLX-200 to one of the key lysosomal storage disorders which have clear unmet medical needs. Furthermore, this designation validates the rationale for clinical use of PLX-200 in GM2 gangliosidoses patients. Our team is poised to initiate a clinical study to confirm its efficacy in GM2 gangliosidoses," 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 "We are committed to provide innovative and patient-friendly drugs to treat a number of rare lysosomal storage disorders, including but not limited to Batten, Niemann-Pick and now Tay-Sachs diseases. With the completion of this milestone, we have comprehensively seized critical regulatory designations to efficiently move forward with the commercialization in lysosomal storage disorders."
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