XORTX Submits Orphan Drug Designation Application to the European Medicines Agency (EMA) to Treat Progressive Kidney Disease

● XORTX’s Proprietary Formulation of Oxypurinol – XORLO – to Treat Progressive Kidney Disease Associated with Autosomal Dominant Polycystic Kidney Disease (ADPKD) ●

CALGARY, Alberta, Aug. 29, 2023 (GLOBE NEWSWIRE) -- XORTX Therapeutics Inc. (“XORTX” or the “Company”) (NASDAQ: XRTX | TSXV: XRTX | Frankfurt: ANU), a late-stage clinical pharmaceutical company focused on developing innovative therapies to treat progressive kidney disease, announces that it has submitted an Orphan Drug Designation application for XORLO to the European Medicines Agency (the “EMA”). The “orphan-drug designation request is for the use of XORTX’s patented unique proprietary formulation of oxypurinol – XORLO – for the treatment of autosomal dominant polycystic kidney disease (ADPKD)”.

The orphan drug designation process (“ODD”) initiated with the submission of this application and is made to the EMA’s COMP (Committee for Orphan Medicinal Products) office. To support this application, a focused data package was provided that included: a review of the basic science related to the mechanism of injury associated with aberrant purine metabolism and hyperuricemia as well as the evidence that XORLO attenuates the accelerating effect of kidney injury, analysis of the number of European patients with ADPKD and arguments to support the likelihood that the new therapy, XORLO, will provide significant, clinically meaningful benefit compared with existing treatment. The EMA’s COMP office, will review this initial application package and provide feedback and a decision, which is expected in December of this year.

Dr. Allen Davidoff, CEO of XORTX, stated, “This EMA ODD submission represents a key milestone for the Company regarding new and existing discoveries made by XORTX and its novel approach to slowing progression of kidney disease in ADPKD. It also follows on receipt of ODD status granted by the U.S. Food and Drug Administration in April 2023. Further updates will be provided once the EMA COMP renders its decision.”

Benefits of EMA Orphan Designation include: Reduced fees for protocol assistance, market authorization applications and annual fees for authorized medicines; Automatic access to centralized procedure for EMA marketing authorization, access to research grants, a simplified approval process and 10 years of market exclusivity. Further information regarding the incentives for orphan designation are available at: https://www.ema.europa.eu/en/human-regulatory/research-development/orp ...