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BioMarin Files BLA for Phenylketonuria Candidate Pegvaliase

BioMarin Pharmaceutical Inc. BMRN announced that it has filed a biologics license application (BLA) with the FDA for its pipeline candidate, pegvaliase for phenylketonuria (PKU). The company had earlier stated that it will file the application in the second quarter of 2017.

BioMarin is looking to get pegvaliase approved for lowering blood phenylalanine (Phe) levels in adult patients with PKU, a rare genetic enzyme deficiency disorder, who have uncontrolled blood Phe levels with existing treatment options.

An application in the EU is expected to be filed by end of the year.

BioMarin’s shares are up 9.6% so far this year, which compares favorably with an increase of 6.4% registered by the Zacks classified Biomed/Genetics industry during this period.

According to information provided by the company, approximately 50,000 individuals in the developed countries are diagnosed with PKU.

We note that BioMarin currently has an approved drug, Kuvan, for this indication in its kitty. Kuvan revenues rose 19% year over year to $92 million in the last reported quarter, first quarter of 2017.

BioMarin acquired all global rights to Kuvan, excluding Japan, and pegvaliase from Merck Serono in Jan 2016.

We remind investors that pegvaliase’s development/regulatory process has not exactly been smooth. The drug has faced several delays and setbacks, which makes the latest announcement a big positive for BioMarin.

In March last year, BioMarin announced results from the phase III study, PRISM-2, on pegvaliase. The study, despite meeting the primary endpoint, failed to achieve the secondary endpoint.

Pegvaliase, if approved, could strengthen BioMarin’s commercial leadership for the treatment of PKU, which can drive company's revenues going forward.

BioMarin carries a Zacks Rank #3 (Hold). Better-ranked stocks in the sector include Regeneron Pharmaceuticals, Inc. REGN, MEI Pharma, Inc. MEIP and VIVUS, Inc. VVUS, all sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Regeneron’s earnings estimates have risen 3.4% for 2017 and 11% for 2018 over the last 60 days. Shares of the company have risen 33.8% so far this year.

VIVUS’ loss per share estimates narrowed 22% for 2017 in the last 60 days. The company delivered positive earnings surprises in all four trailing quarters with an average beat of 233.69%.  Shares of VIVUS have risen 6.1% this year so far.

MEI Pharmahas delivered positive earnings surprises in three of the four trailing quarters with an average beat of 66.56%. Shares have risen 66% this year so far.

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Regeneron Pharmaceuticals, Inc. (REGN): Free Stock Analysis Report
 
VIVUS, Inc. (VVUS): Free Stock Analysis Report
 
BioMarin Pharmaceutical Inc. (BMRN): Free Stock Analysis Report
 
MEI Pharma, Inc. (MEIP): Free Stock Analysis Report
 
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