" class="no-js "lang="en-US"> XORTX Announces Orphan Drug Designation for Kidney Disease
Friday, June 09, 2023

XORTX Announces Receipt of FDA Orphan Drug Designation to Treat Autosomal Dominant Kidney Disease

XORTX Therapeutics, a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, is pleased to announce the grant of Orphan Drug Designation for oxypurinol – “orphan-drug designation request of oxypurinol is granted for treatment of autosomal dominant polycystic kidney disease”.

The orphan drug designation process (“ODD”) begins with an application to the US Food and Drug Administration (“FDA”) Orphan Drug Designation office, and is accompanied by a comprehensive package that includes the basic science, analysis of addressable number of individuals with autosomal dominant polycystic kidney disease (“ADPKD”) and description of the patients projected to be helped by a therapy. The FDA ODD office review of the application package provided by XORTX determined that the evidence that aberrant purine metabolism and high uric acid levels suggest a mechanism of injury in ADPKD and importantly, that the XORLOTM therapy may slow progression of this form of injury.

While this is not approval of XORLOTM for use in patients at the present time, it is a key milestone regarding the new and existing discoveries made by XORTX and regarding a novel approach to slowing progression of kidney disease in ADPDK.

Dr. Allen Davidoff stated, “Orphan drug designation represents a major milestone for the Company in pursuit of marketing approval for XORLOTM, our proprietary formulation of oxypurinol, and the XRx-008 program for ADPKD. This designation was a significant and critical milestone for all of the staff and management at the Company. Further our potential partners indicated that it was a critical requirement underpinning their consideration of the XORLOTM program. We look forward to our upcoming meeting with the FDA on May 1, 2023 to discuss our planned phase 3 clinical program for XORLOTM.”

Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the US) are eligible to apply for ODD, which provides multiple benefits to the sponsor during development and after approval. XORTX intends to pursue these benefits as part of the drug development for XRx-008 for treatment of ADPKD.

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