Ultragenyx Pharma (RARE) Reports Withdrawal of MAA for Aceneuramic Acid Prolonged Release (Ace-ER) in EU

November 11, 2016 8:31 AM EST

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Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced that is has withdrawn its conditional Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) for Aceneuramic Acid Prolonged Release (Ace-ER) for the treatment of adult patients with GNE Myopathy.

The conditional MAA was based on data from the company’s Phase 2 study. 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 Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We are committed to bringing this potential therapy to patients affected by this progressive and debilitating muscle disease.”



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