FDA grants PXL770 orphan drug designation for autosomal-dominant polycystic kidney disease
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The FDA has granted orphan drug designation to PXL770, a first-in-class direct adenosine monophosphate-activated protein kinase activator designed by Poxel to treat autosomal-dominant polycystic kidney disease, according to a press release.
According to the release, this designation follows the completion of preclinical studies that confirmed strategic focus for PXL770 in rare metabolic diseases. Results from the studies were presented at two recent meetings.
“As we have decided to focus PXL770 development efforts exclusively in rare diseases on the basis of our promising data, this orphan drug designation for PXL770 in autosomal-dominant polycystic kidney disease (ADPKD) further strengthens the value of this phase 2 ready program,” Thomas Kuhn, CEO of Poxel, said in the release. “ADPKD is an inherited form of chronic kidney disease where the unmet need is high, with only one approved drug which is associated with significant safety-tolerability challenges. The pathophysiology of ADPKD involves altered kidney metabolism and there is a strong rationale for adenosine monophosphate-activated protein kinase activation as a therapeutic approach. This summer, we completed the preclinical assessment of PXL770 in ADPKD demonstrating compelling benefits of strong suppression of cyst formation in vitro and in vivo and presented these results at recent scientific meetings. We have initiated development program planning and further regulatory interactions.”
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