Ultragenyx's RhGUS Gets FDA Nod For Rare Genetic Disease

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced that the FDA has approved its genetic disorder drug, vestronidase alfa or rhGUS, for treatment of children and adults with mucopolysaccharidosis VII (MPS VII, also known as Sly syndrome). It is approved under the trade name, MEPSEVII.

Notably, mucopolysaccharidosis VII is a progressive and debilitating rare genetic disorder. While rhGUS is the first approved drug for treating this disease.

Shares of Ultragenyx have increased more than 1% in after-hours trading on Nov 16. However, shares of the company have underperformed the industry so far this year. The stock has lost 31.4% in contrast to the broader industry’s increase of 1.7% during the period.

It is important to note that the approval was supported by data from a phase III study, designed to evaluate the safety and efficacy of rhGUS in patients (n=12) aged between 5 and 35 years for MPS 7, a rare lysosomal storage disease. In July 2016, the company announced positive top-line data from the study. Treatment-emergent adverse events were generally mild to moderate in severity.

We remind investors that Ultragenyx submitted a biologics license application ("BLA") to the FDA and a marketing authorization application ("MAA") to the EU earlier in May. Subsequently, the FDA had accepted the company’s BLA on a priority review basis and the company's MAA was also accepted by the EU. Additionally, an opinion from the Committee for Medicinal Products for Human Use (CHMP) is awaited in the first half of 2018.

We note that MPS VII represents an underserved market with no approved therapies yet available to treat the disease. Almost 200 patients across the world are afflicted with this disease, one of the rarest forms of MPS. Hence, the approval and successful commercialization of MEPSE VII will thus be a major boost to the company.

Drugs approved for curing other MPS disorders include BioMarin Pharmaceutical Inc.'s (NASDAQ:BMRN) Aldurazyme (MPS I), Naglazyme (MPS VI) and Vimizim (Morquio A Syndrome (MPS IVA) and Shire plc's (NASDAQ:SHPG) Elaprase (Hunter syndrome (also called MPS-II).

Zacks Rank & Key Picks

Ultragenyx carries a Zacks Rank #3 (Hold). A better-ranked stock in the health care sector is Ligand Pharmaceuticals Inc. (NASDAQ:LGND) , holding a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Ligand's earnings per share estimates have moved up from $3.68 to $3.70 for 2018 over the last 60 days. The company delivered positive earnings surprises in two of the trailing four quarters with an average beat of 8.22%. Share price of the company has surged 39.4% year to date.

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