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BioMarin's Palynziq Gets FDA Approval for Phenylketonuria

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BioMarin Pharmaceutical Inc. (BMRN - Free Report) announced that the FDA has approved its enzyme therapy, Palynziq (pegvaliase). The therapy will lower blood phenylalanine (Phe) levels in adult phenylketonuria (“PKU”) patients with uncontrolled blood Phe levels with existing treatment options.

The approval comes on the back of encouraging data from a phase III study – PRISM-2 – which showed that the drug significantly and substantially reduced blood Phe level compared to placebo.

Palynziq is the first enzyme therapy approved for the treatment of PKU in the United States. The drug will complement sales of Kuvan, also approved for PKU, but has lost exclusivity in the country. The company plans to launch the drug in June this year through a restricted program under a Risk Evaluation and Mitigation Strategy (“REMS”) called Palynziq REMS

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

Shares of the company were up 3.1% in after-hours trading following the announcement. BioMarin’s shares have lost 1% so far this year, which compares favorably with a decrease of 9.8% registered by the industry.


PKU is a rare genetic enzyme deficiency disorder that manifests at birth and if left untreated causes high levels of Phe, which leads to a variety of cumulative toxic effects on the brain. According to information provided by the company, approximately 50,000 individuals in the developed countries are diagnosed with PKU.

We note that Kuvan registered sales of $99.1 million in the first quarter of 2018, a 6.8% rise from the year-ago period. We expect Palynziq to strengthen BioMarin’s commercial leadership for the treatment of PKU, which can drive the company's revenues going forward.

BioMarin also has two late-stage candidates in its pipeline, valoctocogene roxaparvovec for Hemophilia A and vosoritide for Achondroplasia.

Zacks Rank & Key Picks

BioMarin currently carries a Zacks Rank #2 (Buy).

Some other top-ranked stocks to consider in the pharma sector include Aeglea BioTherapeutics, Inc. (AGLE - Free Report) , ANI Pharmaceuticals, Inc. (ANIP - Free Report) and Athersys, Inc. (ATHX - Free Report) . All the three stocks carry a Zacks Rank #2. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Aeglea’s loss estimates narrowed from $1.93 to $1.67 for 2018 and from $3.86 to $3.57 for 2019 over the last 30 days. The company delivered positive earnings surprise in three of the trailing four quarters with an average beat of 19.32%. Its share price has increased 79.7% so far this year.

ANI Pharmaceuticals’earnings estimates increased from $5.54 to $5.79 for 2018 and from $5.72 to $5.80 for 2019 over the last 30 days. The company came up with a positive earnings surprise in three of the four trailing quarters with an average beat of 8.69%.

Athersys’ loss per share estimates narrowed from 34 cents to 24 cents for 2018 and from 51 cents to 41 cents for 2019 over the last 30 days. The stock has gained 35.3% so far this year.

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