Posted by Michael Wonder on 24 May 2017
Ultragenyx announces recombinant human beta-glucuronidase biologics license application and marketing authorisation application filed and accepted for review; FDA grants priority review status
23 May 2017 - Ultragenyx today announced that a biologics license application submitted to the U.S. FDA and a marketing authorisation application submitted to the EMA, for recombinant human beta-glucuronidase (rhGUS, UX003), an investigational therapy for the treatment of mucopolysaccharidosis VII (MPS VII, Sly syndrome) were accepted for review.
The Prescription Drug User Fee Act goal date for a decision is 16 November 2017 and an opinion from the CHMP is expected in the first half of 2018. rhGUS is an enzyme replacement therapy for the treatment of MPS VII.
The FDA granted rhGUS priority review status, which is available for drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists. rhGUS was previously granted orphan drug designation by the FDA.
