RARE Daily

FDA Grants Fast Track Designation to Poxel’s Experimental PXL065 for X-linked Adrenoleukodystrophy

February 17, 2022

The U.S. Food and Drug Administration has granted Fast Track designation to Poxel’s PXL065 for the treatment of patients with adrenomyeloneuropathy, the most common form of X-linked adrenoleukodystrophy (ALD).

Photo: Thomas Kuhn, CEO of Poxel

PXL065 is a novel, proprietary deuterium-stabilized R-stereoisomer of pioglitazone that is preparing to enter a phase 2a clinical proof-of-concept biomarker study midyear.

X-linked adrenoleukodystrophy (ALD) is a rare neurometabolic disease caused by mutations in the ABCD1 gene which encodes for a key protein that is required for metabolism of very long chain fatty acids (VLCFA) by peroxisomes (cellular organelles). ALD is the most common leukodystrophy with a prevalence of about 1 in 10,000 individuals in the general population. Adrenomyeloneuropathy (AMN) is the most common form of ALD that typically occurs in adolescence through adulthood. AMN is characterized by chronic and progressive distal axonopathy involving the long tracts of the spinal cord and to a lesser extent the peripheral nerves resulting in progressive stiffness and weakness in the legs, impaired gait and balance, incontinence, and loss of sensation. Nearly all men with a diagnosis of ALD will develop AMN, and many women also present with features of AMN with a later onset. There are no approved medicines for ALD (other than glucocorticoid supplements for associated adrenal insufficiency).

“By awarding Fast Track designation to PXL065, the FDA recognizes the drug’s potential to address a significant unmet medical need for patients with ALD, where no approved therapies currently exist,” said Thomas Kuhn, CEO of Poxel. “This is a very powerful acknowledgement and good news for patients waiting to be treated.”

Fast Track designation may be awarded by the FDA to investigational drugs that treat a serious or life-threatening condition and provide a therapy where none exists or provide a therapy that may be potentially better than available therapy. The program provides enhanced access to the FDA, with regular and more frequent opportunities for consultation and discussion. In addition, drugs with Fast Track designation may be eligible for Accelerated Approval, in which a new medicine is approved prior to the availability of definitive data, and Priority Review, in which the standard 10-month review process is reduced to six months. Fast Track drugs may also enter a ‘rolling review’ of their NDA submission, in which sections are submitted and reviewed as they become available, substantially expediting the approval process.

Author: Rare Daily Staff

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