Ultragenyx (RARE) Focuses On Pipeline Amid Low Revenues

 | Aug 23, 2019 07:36AM ET

We issued an updated report on Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) on Aug 22.

The company has two approved therapies in its portfolio. Mepsevii (vestronidase alfa) was approved in the United States, in November 2017, for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. The drug is under review in the EU for the same indication. Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 (FGF23), is approved in the United States for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients. The drug received European conditional marketing authorization, in February 2018, for the treatment of XLH with radiographic evidence of bone disease in children one year of age or older, and adolescents with growing skeletons. A filing for adults with XLH is also planned in Europe.

Shares of the company increased 31.1% year to date against the Zacks Investment Research

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