The Food and Drug Administration (FDA) has granted Fast Track designation to PXL770 for the treatment of patients with adrenomyeloneuropathy.
Adrenomyeloneuropathy (AMN) is the most common form of X-linked adrenoleukodystrophy (ALD). It is caused by mutations in the ABCD1 gene, which is required for the metabolism of very long fatty acids (VLCFA) by peroxisomes, resulting in elevated VLCFA levels.
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.
PXL770 is a novel, first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator. In preclinical studies, PXL770 was found to suppress elevated VLCFA levels in ALD patient derived cells.
The Company also recently received Fast Track designation for another AMN investigational agent, PXL065, a novel, proprietary deuterium-stabilized R-stereoisomer of pioglitazone.
“We are preparing the next steps to initiate our two phase 2a clinical studies midyear, with results anticipated early 2023,” said Poxel CEO, Thomas Kuhn.
- Poxel announces PXL770 awarded FDA Fast Track designation for X-linked adrenoleukodystrophy. News release. Poxel SA. April 11, 2022. https://www.poxelpharma.com/en_us/news-media/press-releases/detail/209/poxel-announces-pxl770-awarded-fda-fast-track-designation
- Poxel announces PXL065 granted FDA Fast Track designation for X-linked adrenoleukodystrophy. News release. Poxel SA. February 16, 2022. https://www.poxelpharma.com/en_us/news-media/press-releases/detail/205/poxel-announces-pxl065-granted-fda-fast-track-designation
- Pipeline: Rare Diseases-ALD. Poxel SA. Accessed April 12, 2022. https://www.poxelpharma.com/en_us/pipeline/rare-diseases-ald
Source: Medical Bag https://www.medicalbag.com/home/news/ampk-activator-gets-fast-track-status-for-x-linked-adrenoleukodystrophy/