XORTX applies to EMA for orphan drug designation for PKD therapeutic


XORTX Therapeutics, a late-stage clinical pharmaceutical company focused on developing “innovative therapies to treat progressive kidney disease”, has announced in a press release that it has submitted an orphan drug designation (ODD) application for Xorlo to the European Medicines Agency (EMA). The ODD 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 release states.

The ODD process is initiated with the submission of this application and is made to the EMA’s Committee for Orphan Medicinal Products (COMP) 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 hyperuricaemia 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 2023.

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 US Food and Drug Administration (FDA) in April 2023. Further updates will be provided once the EMA COMP renders its decision.”

Benefits of EMA ODD include reduced fees for protocol assistance, market authorisation applications and annual fees for authorised medicines, and automatic access to centralised procedure for EMA marketing authorisation. It also provides access to research grants, a simplified approval process and 10 years of market exclusivity, the press release concludes.


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