Ultragenyx seeks marketing authorization for Ace-ER tablets in Europe

Ace-ER is designed for the replacement of the deficient sialic acid substrate in patients with GNE Myopathy.

A defect in the biosynthetic pathway for sialic acid, with onset usually occurring in the twenties, causes GNE Myopathy, which is also known as hereditary inclusion body myopathy (HIBM).

It is a rare, severe, progressive, genetic neuromuscular disease. There are about 2,000 GNE Myopathy patients in the developed world with no approved therapy at present.

Ultragenyx carried out a Phase 2 randomized, double-blind, placebo-controlled clinical study. A decision from the European Commission would be anticipated in the second half of next year after a positive opinion from the Committee for Medicinal Products for Human Use (CHMP).

In May 2015, the company started a phase 3 global, randomized, double-blind, placebo-controlled clinical study of Ace-ER in about 80 patients. It is continuing to enroll patients.

The primary endpoint of the study is upper extremity muscle strength.

Ultragenyx Pharmaceutical CEO Emil Kakkis said: "We have reached an important milestone for both Ultragenyx and patients living with GNE Myopathy.

"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 licensed rights to aceneuramic acid extended release and its potential use in treating GNE Myopathy from Nobelpharma, AAI Pharma, and the HIBM Research Group.