Wednesday, August 30, 2023
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/orphan-designation/orphan-incentives
About the XRx-008 Program
Oxypurinol is a xanthine oxidase inhibitor (“XOI”) with important pharmacologic characteristics ideal for administration to individuals with ADPKD. Key pharmacologic attributes include:
1/ The ability to act in the circulation, kidney and cardiovascular tissue and inhibit the production of uric acid thereby attenuate the related mechanism of injury whereby xanthine oxidase accelerates the progression of renal and cardiac diseases.
2/ XORLO™ provides substantially increased absorption of oxypurinol. This approach provides an effective, well tolerated drug with an extensive clinical safety experience suggesting the Company’s XRx-008 program has the capacity to provide an XOI with a superior product profile indicated to slow the accelerating decline in kidney function in ADPKD patients.
About ADPKD
ADPKD is a rare disease that affects more that 10 million individuals worldwide.1,2 ADPKD is typically diagnosed based upon expansion of fluid-filled cysts in the kidneys. Over time, the increasing number and size of cysts can contribute to structural and functional changes to kidneys and is frequently accompanied by chronic pain which is a common problem for patients with ADPKD.3 Expansion of cysts is thought to compress healthy functioning tissue surrounding the cysts and contribute to further loss of kidney function, fibrosis, impaired nutrient exchange and impaired kidney function, leading ultimately to end-stage renal disease.1 For individuals with progressing ADPKD, treatment recommendations include anti-hypertensive treatment, dietary restrictions, and, for a limited percentage of suitable patients who can tolerated ADPKD specific pharmacotherapy.4 New, more broadly applicable therapies to effectively slow decline of kidney function in ADPKD are needed.
