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Ultragenyx's (RARE) KRN23 Accepted for Review in the EU

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Ultragenyx Pharmaceutical Inc. (RARE - Free Report) announced that the European Medicines Agency (EMA) has accepted to review the company’s Marketing Authorization Application (MAA) for KRN23 for the treatment of X-linked hypophosphatemia (XLH).

KRN23, an antibody targeting fibroblast growth factor 23 (FGF23), is being developed in collaboration with Kyowa Hakko Kirin Co., Ltd.

Note that Ultragenyx’s share price movement in 2016 shows that the company has outperformed the Zacks classified Medical-Biomedical/Genetics industry. Specifically, the stock lost 18.7% during this period, compared to the industry’s plunge of 20.5%.



Coming back to the latest news, the company expects an opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP) in the second half of 2017.

Meanwhile, a biologics license application (BLA) for KRN23 is expected to be filed in the U.S. in the second half of 2017. The companies are in discussions with the FDA for the details of the planned submission.

We remind investors that in Jun 2016, the FDA had granted breakthrough therapy designation to KRN23 for the treatment of XLH in pediatric patients aged one year and above.

Currently, KRN23 is being evaluated in a phase III study in adults with XLH. Data from the study are expected to be out in the first half of 2017. KRN23 is also in a phase II and phase III study for the treatment of pediatric patients.

Apart from XLH, KRN23 is also being developed in a phase II study for the treatment of tumor-induced osteomalacia (TIO).

We note that Ultragenyx is making impressive progress with its pipeline candidates. The company expects to submit regulatory filings for rhGUS (UX003) for the treatment of mucopolysaccharidosis 7 (MPS 7) in both the U.S. and the EU in the first half of 2017.

Going forward, we expect investors to focus on the CHMP’s opinion on KRN23 and the company’s regulatory filing for rhGUS.

ULTRAGENYX PHAR Price and Consensus

 

ULTRAGENYX PHAR Price and Consensus | ULTRAGENYX PHAR Quote

Ultragenyx currently carries a Zacks Rank #4 (Sell).

Key Picks in the Sector

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Cambrex’s earnings estimates increased 3.7% for 2016 and 5.2% for 2017 in the last 60 days. The company posted a positive earnings surprise in three of the four trailing quarters, with an average beat of 19.78%. Its share price was up 26.7% in the past one year.

Arbutus’ loss estimates narrowed from $2.15 to $1.71 for 2016 and from $1.96 to $1.52 for 2017 in the last 60 days. The company posted a positive surprise thrice in the four trailing quarters with an average beat of 59.31%.

Anika’s earnings estimates for 2016 and 2017 were up 3.9% and 0.5% respectively for the last 60 days. The company recorded a positive earnings surprise in each of the last four quarters, the average being 33.14%. Its share price was up 35.8% in the past one year.

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