Poxel Receives Orphan Drug Designation from the European Commission for PXL770 and PXL065 for Treatment of Adrenoleukodystrophy
POXEL SA, a clinical-stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare metabolic disorders, has announced that European Commission has granted orphan drug designation (ODD) for PXL770 and PXL065 for the treatment of adrenoleukodystrophy (ALD). The decision follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA).
The U.S. Food and Drug Administration has previously granted ODD and Fast Track Designation to both PXL770 and PXL065 for the treatment of ALD. These molecules have separate and distinct mechanisms of action. PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator. PXL065 is a novel, proprietary deuterium-stabilized R-stereoisomer of pioglitazone which exerts effects via multiple non-genomic pathways engaged by thiazolidinedione molecules. Both compounds are preparing to enter into Phase 2a clinical Proof-of-Concept (POC) biomarker studies in ALD patients with adrenomyeloneuropathy (AMN) as soon as possible, subject to financing.
“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”, noted Thomas Kuhn, CEO of Poxel. “We are in active discussions to restructure our current debt obligations and secure the funding to execute our strategy in rare metabolic diseases”.
ODD in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medical Products (COMP). To qualify for ODD from the European Commission, a product candidate must be intended to treat, prevent, or diagnose a life-threatening or chronically debilitating disease that does not affect more than 5 in 10,000 people across the EU. In addition, there must be sufficient clinical or non-clinical data to suggest the product candidate may produce clinically relevant outcomes, and grounds to indicate it can provide a significant benefit over any currently authorized products. Receiving an orphan drug designation from the European Commission provides companies with certain benefits and incentives including clinical protocol assistance, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees, and ten years of market exclusivity upon receipt of marketing authorization in the EU. The availability of market exclusivity is intended to encourage the development of medicines for rare diseases by protecting them from competition from similar medicines with similar indications, which cannot be marketed during the exclusivity period.
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