Drug Development

Ultragenyx nixes plan to seek quick OK for rare disease drug in Europe

Emil D. Kakkis, Ultragenyx

Emil D. Kakkis, Ultragenyx

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.

The best place to read Endpoints News? In your inbox.

Full-text daily reports for those who discover, develop, and market drugs. Join 21,000+ biopharma pros who read Endpoints News by email every day.

Free Subscription