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Ultragenyx Granted Orphan Designations in Europe for Two Lead Product Candidates, UX001 for HIBM and UX003 for MPS 7

NOVATO, Calif., March 28, 2012 /PRNewswire/ -- 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.

About HIBM  

HIBM is also known as GNE myopathy, distal myopathy with rimmed vacuoles (DMRV) and Nonaka disease.  HIBM is a severe, adult-onset, progressive, genetic neuromuscular disease caused by a deficiency of an enzyme in the first step of sialic acid biosynthesis needed for the modification of proteins and fats.  Patients with HIBM typically begin to have weakness and abnormal walking at 18 to 30 years of age.  Over the ensuing 10 to 20 years, many patients progressively lose significant functional ability and become wheelchair-bound.  There are no current treatments for this disease.

About MPS 7

Mucopolysaccharidosis type 7 (MPS 7), also known as Sly syndrome, is a rare genetic, metabolic disorder.  MPS 7 is caused by the deficiency of beta-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans (GAGs) dermatan sulfate and heparan sulfate.  These complex GAG carbohydrates are a critical component of many tissues. The inability to properly breakdown GAGs leads to a progressive accumulation in many tissues and multi-system disease.  There are a wide variety of clinical symptoms including enlarged organs, stiff joints, respiratory disease and cardiac complications.  There are no approved therapies for MPS 7 today.

About Ultragenyx

Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan and ultra-orphan diseases, to market.  The company focuses on rare metabolic diseases that affect small numbers of patients, but for which the unmet medical need is high and there are no effective treatments.  Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these underserved diseases.  Ultragenyx' lead program, UX001, is being evaluated as a potential treatment for hereditary inclusion body myopathy (HIBM), also known as GNE myopathy.  The UX001 program has been granted orphan drug designation in the US and the EU.

The company is led by an experienced management team in rare disease therapeutics.  Ultragenyx is striving toward an improved model for successful rare disease drug development which has the potential to increase efficiency while maintaining appropriate safety and efficacy standards. The company believes that it can deliver significant value to patients by building a diverse and high quality pipeline of rare disease therapeutics and efficiently transforming good science into great medicine.

For more information on Ultragenyx, please visit the company's website at




SOURCE Ultragenyx
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