ARTICLE | Company News

AZ gets Wellstat's voucher after ultra-Orphan approval

September 9, 2015 1:12 AM UTC

AstraZeneca plc (LSE:AZN, NYSE:AZN) will obtain a Priority Review voucher from Wellstat Therapeutics Corp. (Gaithersburg, Md.) for an undisclosed sum. Wellstat received the voucher under FDA's rare pediatric disease Priority Review voucher program following the Friday approval of Xuriden uridine triacetate ( PN401) to treat hereditary orotic aciduria, a metabolic disorder the agency said affects about 20 people worldwide. The compound had been under Priority Review, and has Orphan Drug and breakthrough therapy designations.

FDA based its approval primarily on a four-patient study in which the drug stabilized pre-specified hematologic parameters. Wellstat VP of R&D Michael Bamat told BioCentury that FDA encouraged the company to submit an NDA and to use the voucher program. ...