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Why Is Orphan Drug Company Ultragenyx Pharmaceutical In The News ?

December 20, 2012

       Per a December 20, 2012 News Release, Ultragenyx Pharmaceutical, a biotechnology company launched just 2 1/2 years ago, announces the completion of a $75 million Series B round financing. According to, the financing is “ ….. one of the largest private placements of the year for a biotech company”.  Ultragenyx Pharmaceutical’s plan is an initial public offering in 2014 and products to be sold starting in 2016.

       Ultragenyx Pharmaceutical develops therapeutics for rare and ultra-rare diseases. Ultragenyx Pharmaceutical’s founder, CEO, and President is Emil Kakkis, M.D., Ph.D.  During Dr. Kakkis’s tenure at BioMarin Pharmaceutical, 3 orphan drugs (Aldurazyme for MPS I, Naglazyme for MPS VI, and Kuvan for PKU) were developed and approved.

        Orphan Druganaut Blog reported in August 2012, on Ultragenyx Pharmaceutical’s raising of $15 million for a potential 1st treatment of Hereditary Inclusion Body Myopathy (HIBM). HIBM is a rare, progressive neuromuscular disease caused by a deficiency in sialic acid that results in muscles slowly wasting away. Many patients require a wheelchair within 10 – 15 years of disease onset.

       Ultragenyx Pharmaceutical’s orphan drug UX001 (Sialic Acid) receives FDA Orphan Drug Designation (ODD) on September 23, 2011, for the treatment of HIBM. The oral drug UX001 uses an extended-release formulation of sialic acid.

       The Internet buzz about Ultragenyx Pharmaceutical’s $75 million financing :

1) December 20, 2012 article titled, “Ültragenyx, Led By BioMarin Vet, Snags $75M for Ultra-Rare Diseases”

2) December 20, 2012 article titled, “Investors gamble $75M on Ultragenyx’s plan to jumpstart rare-disease player”

3)  San Francisco Business Times December 20, 2012 article titled, “Ultragenyx raises $75 Million to push ‘Lazarus-like’ 3rd drug into mid-stage study”.


Advancement of Research for Myopathies (ARM)

Neuromuscular Disease Foundation (NDF)

Ultragenyx Pharmaceutical Pipeline

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