Ultragenyx withdraws MAA for HIBM therapy

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said Friday that it withdrew an MAA for Ace-ER (UX001) to treat GNE myopathy, a muscle-wasting disorder also known as hereditary inclusion body myopathy. The company had sought conditional approval of the product in the EU based on Phase II data (see BioCentury Extra, Oct. 2, 2015).

According to the company, EMA’s CHMP said the Phase II data were insufficient to support Ace-ER's approval. Ultragenyx intends to submit an MAA for full approval after it receives data in 2H17 from an ongoing Phase III trial of Ace-ER...