FDA grants Fast Track Designation to rare disease treatment 

The US Food and Drug Administration (FDA) has granted Fast Track Designation to a treatment for the rare paediatric disease late-infantile neuronal ceroid lipofuscinosis (LINCL/CLN2 disease). 

PLX-200, developed by biotechnology company Polaryx Therapeutics, will now undergo an expedited review with the potential to address the paediatric disease with unmet need. 

The treatment is an orally available compound comprised of gemfibrozil, an FDA-approved lipid regulating agent in the fibrate family. Preclinical trials show gemfibrozil can cross the blood-brain barrier, being considered safe for use in adults over several decades of clinical investigation. 

“Receiving Fast Track Designation represents an important regulatory milestone as we prepare to initiate the SOTERIA Phase II basket trial evaluating PLX200 across multiple lysosomal storage disorders” said Alex Yang, Chair and Chief Executive Officer of Polaryx Therapeutics.  

“We appreciate the FDA’s recognition of the potential of PLX200 to address the significant unmet medical need in CLN2 disease and look forward to continued engagement with the agency as we advance development of this programme.” 

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