Faced with a pushback from European regulators, Ultragenyx today says that it is jerking its application for a conditional approval of its drug for GNE Myopathy.
Ultragenyx had hoped to score an early OK on Phase II data for Aceneuramic Acid Prolonged Release (Ace-ER). But regulators shook their heads on that idea. Now the Novato, CA-based rare disease specialist says it plans to regroup and go at this again with late-stage data in H2 2017.
Shares of Ultragenyx slid 5% in pre-market trading.
According to the biotech:
During the Committee for Medicinal Products for Human Use (CHMP) meeting, the CHMP indicated that the Phase 2 study was encouraging but did not provide a sufficient amount of evidence to support an approval at this time. Ultragenyx intends to obtain additional efficacy data from its fully-enrolled global phase 3 study to confirm the effects of Ace-ER, and plans to submit an MAA for full approval after data from this study are available in the second half of 2017.
“Our Phase 3 study is on track and is designed to confirm the encouraging results seen in the Phase 2 study through a larger trial with a primary endpoint and a placebo period that lasts for the full 48-week duration of the study,” said Ultragenyx CEO Emil D. Kakkis.
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