EMA to review Ultragenyx's Ace-ER to treat HIBM

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said EMA accepted for review an MAA for Ace-ER ( UX001) to treat GNE myopathy, a muscle-wasting disorder also known as hereditary inclusion body myopathy.

The company is seeking conditional approval for Ace-ER based on Phase II data. It expects data in early 2017 from an ongoing Phase III trial of the extended-release oral formulation of aceneuramic acid. The Phase III trial is designed to satisfy EMA's requirement for a confirmatory trial and to serve as the basis for an NDA submission. President and CEO Emil Kakkis declined to give a timeline for the submission. ...