Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the European Medicines Agency (EMA) has granted orphan drug designations for sialic acid for the treatment of hereditary inclusion body myopathy (HIBM) and recombinant human beta-glucuronidase for the treatment of mucopolysaccharidosis type 7 (MPS 7). Orphan designation in the European Union provides several benefits including 10 years of market exclusivity post-authorization, fee reductions, scientific assistance for marketing authorization and access to community marketing authorization.
UX001 is an extended release formulation of sialic acid (SA-ER) intended as a substrate replacement therapy for HIBM, a severe, neuromuscular disease caused by sialic acid deficiency. UX003 is a recombinant human beta-glucuronidase intended as an enzyme replacement therapy for the treatment of MPS 7, an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme beta-glucuronidase.
"Ultragenyx continues to make significant progress in advancing our pipeline of potential treatments for patients affected by ultra-rare diseases," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "I am pleased that our regulatory team was able to obtain orphan designations in both the US and the EU so promptly for both of our lead programs. We will continue to work collaboratively with regulatory authorities as our clinical development programs move forward."
A Phase 2 study for UX001 SA-ER in patients with HIBM is planned to begin enrollment in the second quarter of this year. A Phase 1 study for UX003 is anticipated to begin in the first quarter of 2013. Both UX001 and UX003 have been granted orphan designations by the FDA.