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Keeping Track: FDA Approves Two Novel Orphan Drugs; Breakthrough Applications Keep Agency Busy

 
• By Bridget Silverman

The latest drug development news and highlights from our FDA Performance Tracker.

Orphan drugs were central to FDA’s busy week of approvals, as the agency cleared two novel agents and a new use for a blockbuster biologic for rare disease indications.

FDA approved the first product from privately held Wellstat Therapeutics Corp. on Sept. 4, 2015. Xuriden (uridine triacetate), a new molecular entity pyrimidine analog, is indicated as uridine replacement for treatment of patients with the rare genetic disorder hereditary orotic aciduria (HoA)

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