Ultragenyx Announces Aceneuramic Acid Prolonged Release Marketing Authorization Application Filed and Accepted for Review by European Medicines Agency

Ultragenyx Pharmaceutical Inc. RARE, a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced it has successfully filed a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for aceneuramic acid prolonged release (Ace-ER; UX001) tablets intended for patients with GNE Myopathy. Ace-ER is designed to replace the deficient sialic acid substrate in patients with GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), a rare, progressive muscle-wasting disease. "We have reached an important milestone for both Ultragenyx and patients living with GNE Myopathy," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer of Ultragenyx. "It is the company's first filing for marketing approval, just five years after its founding, and the first marketing application of a potential treatment for patients affected by this progressive debilitating muscle disease." Ultragenyx is seeking to obtain conditional approval from the EMA for Ace-ER for the treatment of adult patients with GNE Myopathy. The MAA submission is based on positive data from a Phase 2 randomized, double-blind, placebo-controlled clinical study. If a positive opinion is received from the Committee for Medicinal Products for Human Use (CHMP), a decision from the European Commission would be expected in the second half of 2016. A Phase 3 global, randomized, double-blind, placebo-controlled clinical study of Ace-ER in approximately 80 patients was initiated in May 2015 and continues to enroll patients. The primary endpoint of the study is upper extremity muscle strength. The study was designed based on feedback from the U.S. Food and Drug Administration (FDA) and the EMA, and data are expected in early 2017. This study is intended to satisfy the EMA's requirement for confirmatory data and serve as the basis for a filing for FDA approval.