FDA Awards Fast Track Designation to Poxel’s PXL770 for X-linked Adrenoleukodystrophy

The U.S. Food and Drug Administration has granted Fast Track Designation to Poxel’s PXL770 for the treatment of patients with adrenomyeloneuropathy, the most common form of X-linked adrenoleukodystrophy.

Photo: Thomas Kuhn, CEO of Poxel

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 up to 1 in 10,000 individuals in the general population. Forms of this disease include cerebral ALD (C-ALD) and adrenomyeloneuropathy (AMN), which is the most common form, and typically occur in adolescence through adulthood. AMN is characterized by chronic and progressive distal axonopathy resulting in progressive stiffness and weakness in the legs, impaired gait and balance, incontinence, and loss of sensation. There are no approved medicines for ALD (other than glucocorticoid supplements for associated adrenal insufficiency). C-ALD when first detected in early childhood, can be treated with hematopoietic stem cell transplantation. HSCT is currently limited to early stage of C-ALD and this procedure is at risk of severe adverse reactions.

PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator that is preparing to enter a phase 2a clinical proof-of-concept biomarker study midyear, subject to additional financing.

“Having PXL770 awarded Fast Track Designation by the FDA soon after our other promising product, PXL065, is a strong recognition of the potential of both our programs in adrenoleukodystrophy, a significant unmet medical need,” said Thomas Kuhn, CEO of Poxel. “Poxel aims at developing innovative treatments to improve the life of patients with serious and rare chronic diseases with metabolic pathophysiology, and the Fast Track Designation could offer PXL770 the ability to substantially accelerate the approval timeline in ALD. We are preparing the next steps to initiate our two phase 2a clinical studies midyear, with results anticipated early 2023.”

Fast Track designaation is designed to expedite development of pharmaceutical products that demonstrate the potential to address unmet medical needs in serious or life-threatening conditions. It allows for substantially enhanced access to FDA, including opportunities for face-to-face meetings and written consultations throughout the remaining development program. Drugs with Fast Track designation are eligible to apply for Accelerated Approval and Priority Review at the time of a New Drug Application submission, which may result in faster product approval, and also allows for ‘rolling review,’ whereby completed sections of the NDA may be submitted as they become available, rather than at the end of development.

The phase 2a clinical proof-of-concept biomarker studies for Poxel’s two products, PXL770 and PXL065, in X-linked ALD are, subject to additional financing, anticipated to begin midyear, with results anticipated early 2023.

Author: Rare Daily Staff