Ultragenyx Receives Priority Review for rhGUS from FDA

Ultragenyx Pharmaceutical Inc. RARE recently announced that it has submitted regulatory applications in both the U.S. and the EU for recombinant human beta-glucuronidase (rhGUS). This is an investigational candidate that is being developed for the treatment of mucopolysaccharidosis VII (MPS VII), also known as Sly syndrome.

Ultragenyx submitted a biologics license application (BLA) to the FDA and a marketing authorization application (MAA) to the EU. The FDA has accepted the company's BLA on a priority review basis and set a PDUFA action date of Nov 16, 2017. Moreover, the company’s MAA was accepted by the EU and an opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in the first half of 2018.

Note that rhGUS was previously granted orphan drug designation by the FDA.

Shares of Ultragenyx have underperformed the Zacks classified Medical-Biomed/Genetics industry in the past one year. The stock has lost 16.1% during the period, while the broader industry witnessed a decrease of 11%.

Coming back to the latest news, the regulatory filings were supported by data from  a phase III study designed to evaluate the safety and efficacy of rhGUS in patients (n=12) aged 5–35 years for MPS 7 – a rare lysosomal storage disease. In Jul 2016, the company announced positive top-line data from the study. Data demonstrated a rapid and sustained reduction of 64.8% from baseline in urinary GAG (dermatan sulfate) excretion after 24 weeks of treatment, thereby meeting the primary endpoint. It also provided evidence of clinical improvement. Treatment-emergent adverse events were generally mild to moderate in severity.

We note that MPS 7 represents an underserved market with no approved therapies presently available to treat the disease. Approval and successful commercialization of rhGUS will thus be a major boost for the company.

Drugs currently approved for the treatment of other MPS disorders include BioMarin Pharmaceutical Inc.’s BMRN Aldurazyme, Naglazyme and Vimizim and Shire plc’s SHPG Elaprase.

Ultragenyx Pharmaceutical Inc. Price

 

Ultragenyx Pharmaceutical Inc. Price | Ultragenyx Pharmaceutical Inc. Quote

Zacks Rank and Key Picks

Ultragenyx currently carries a Zacks Rank #3 (Hold). A better-ranked stock in healthcare sector is VIVUS, Inc. VVUS, which sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

VIVUS’s loss per share estimates narrowed from 50 cents to 39 cents for 2017, over the last 30 days. The company posted positive earnings surprises in all four trailing quarters with an average beat of 233.69%.

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