Wellstat Therapeutics Corporation has announced in September that the US Food and Drug Administration granted marketing approval for XURIDEN™ (uridine triacetate), a pyrimidine analog for uridine replacement indicated for the treatment of hereditary orotic aciduria (HOA). With the approval of XURIDEN for the treatment of HOA, the FDA also granted Wellstat Therapeutics a Rare Pediatric Disease Priority Review Voucher. HOA is an extremely rare, potentially life-threatening, genetic disorder in which patients cannot synthesize adequate amounts of uridine and consequently can suffer from hematologic abnormalities, failure to thrive, a range of developmental delays, and episodes of crystalluria leading to obstructive uropathy.
“The FDA’s dedication to encouraging companies to make treatments available for patients with rare pediatric diseases has been evident throughout the development of XURIDEN, and this approval and voucher grant highlight that commitment.”
“The approval of XURIDEN and the granting of a Rare Pediatric Disease Priority Review Voucher are significant milestones for Wellstat Therapeutics Corporation,” said Samuel J. Wohlstadter, CEO. “The FDA’s dedication to encouraging companies to make treatments available for patients with rare pediatric diseases has been evident throughout the development of XURIDEN, and this approval and voucher grant highlight that commitment.”
Michael K. Bamat, Ph.D., Vice President of Research and Development said, “The FDA’s approval of XURIDEN for hereditary orotic aciduria is an important breakthrough for patients with this rare, potentially life-threatening orphan disease and for their families. There have been no approved treatments for these patients until now.”
In September 2014, Wellstat entered into an agreement with AstraZeneca under which the Pediatric Priority Review Voucher would be transferred to AstraZeneca upon approval. Financial terms of the agreement have not been disclosed.