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Ultragenyx's Rare Disease Drug Mepsevii Gets EU Approval

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Ultragenyx Pharmaceutical Inc. (RARE - Free Report) announced that the European Commission (EC) has approved the Marketing Authorization Application (MAA) for Mepsevii (vestronidase alfa) for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome. 

We remind investors that Mepsevii, an enzyme replacement therapy, was approved in the United States in November 2017 for the treatment of children and adults with MPS VII. It is the first and the only medicine approved for the treatment of children and adults suffering from MPS VII in the United States. 

The European approval follows a positive opinion adopted on Jun 28, 2018 by the European Committee for Medicinal Products for Human Use (CHMP), recommending approval of Mepsevii under exceptional circumstances. The European Medicines Agency (EMA) granted Orphan Drug designation to Mepsevii in March 2012.

Shares of Ultragenyx have increased 69.6% year to date against the industry’s decline of 4.0%.

 

Meanwhile, the company has a number of candidates in its pipeline. It is also looking to expand the label of its approved drug Crysvita (burosumab). Crysvita, 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.

Crysvita 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 and older, and adolescents with growing skeletons. A filing to expand the label in order to include adults with XLH is also planned. It is the only treatment that targets the underlying cause of this rare, hereditary, lifelong disease and can generate significant sales for the company. 

Ultragenyxis also developing a synthetic triglyceride-UX007, which is currently in a phase II study for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). The company is in discussions with the FDA and the EMA, based on positive phase II study data for UX007, to determine the acceptability of its filing based on the totality of currently available data for the treatment of LC-FAOD.

Zacks Rank & Stocks to Consider

Ultragenyx has a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Gilead Sciences Inc. (GILD - Free Report) , Ligand Pharmaceuticals Inc. (LGND - Free Report) and Celgene Corp. (CELG - Free Report) . While Gilead and Ligand sport a Zacks Rank #1 (Strong Buy), Celgene carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Gilead’s earnings per share estimates have increased from $6.11 to $6.57 for 2018 and from $6.36 to $6.48 for 2019 over the past 60 days. The company delivered a positive earnings surprise in three of the trailing four quarters with an average beat of 6.43%. The stock has rallied 4.4% so far this year.

Ligand’s earnings per share estimates moved up from $4.93 to $5.64 for 2018 and from $5.39 to $5.59 for 2019 in the last 30 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 59.5%. Share price of the company has increased 87.6% year to date.

Celgene’s earnings per share estimates have increased from $8.74 to $8.76 for 2018 and from $10.59 to $10.65 for 2019 over the past 30 days. The company delivered a positive earnings surprise in all of the trailing four quarters with an average beat of 2.38%.

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