Policy & Regulation
Poxel announces European Commission grant of orphan drug designation for PXL770 and PXL065 to treat adrenoleukodystrophy
25 January 2023 -

Biopharmaceutical company POXEL SA (Euronext : POXEL - FR0012432516) reported on Wednesday that the European Commission has granted orphan drug designation (ODD) for PXL770 and PXL065 for the treatment of adrenoleukodystrophy (ALD).

The company said that this decision follows a positive opinion from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). ODD and Fast Track Designation to both PXL770 and PXL065 for the treatment of ALD was previously granted by the US Food and Drug Administration.

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

Subject to financing, 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.