Poxel given EC’s Orphan Drug Designation for rare metabolic disorders

pharmafile | January 25, 2023 | News story | Research and Development  

Clinical stage biopharmaceutical company Poxel, who focus on developing innovative treatments for chronic, serious diseases with metabolic pathophysiology, has been awarded Orphan Drug Designation (ODD) for two drugs for the treatment of adrenoleukodystrophy (ALD).


The FDA had previously granted OOD and Fast Track Designation for PXL770 and PXL065, with the EC’s decision coming after a positive indication from the Committee for Orphan Medicinal Products (COMP) of the European’s Medicines Agency (EMA).


PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator. PXL065 is a novel, proprietary deuterium-stabilised R-stereoisomer of pioglitazone which exerts effects via multiple non-genomic pathways engaged by thiazolidinedione molecules.


ALD is an orphan neurometabolic disease caused by mutations in the ABCD1 gene, which encodes for a key protein required for metabolism of very long fatty chain acids (VLFCA) by peroxisomes (cellular organelles). The most common form is adrenomyeloneuropathy (AMN), the symptoms of which include progressive weakness and stiffness in the legs, an impaired gait and balance, incontinence and loss of sensation.


Thomas Kuhn, CEO of Poxel stated: “Orphan Drug Designation in adrenoleukodystrophy for both PXL770 and PXL065 further strengthens the value of these clinical assets where we are preparing to initiate phase 2 proof-of-concept studies, pending additional financing. We are in active discussions to restructure our current debt obligations and secure the funding to execute our strategy in rare metabolic diseases.”


James Spargo

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