UX 001 (aceneuramic acid prolonged release) filed with EU for hereditary inclusion body myopathy- Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical has successfully filed a Marketing Authorization Application with the European Medicines Agency for UX 001 tablets (aceneuramic acid prolonged release) intended for patients with GNE Myopathy. UX 001 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.

The MAA submission is based on positive data from a Phase II 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 III global, randomized, double-blind, placebo-controlled clinical study of UX 001 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 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.