Yahoo Finance BioMarin (BMRN) Gets FDA Approval for Higher Dose of Palynziq
BioMarin Pharmaceutical Inc. BMRN announced that the FDA has approved its supplemental biologics license application (sBLA), seeking to increase the maximum allowable dose of Palynziq (pegvaliase-pqpz) injection to 60 mg for the treatment of adults with Phenylketonuria (PKU).
Palynziq injection, which is indicated to reduce blood phenylalanine (Phe) levels in PKU patients, was approved for 40 mg dose until now. Importantly, 19% of participants in the phase III PRISM studies required a 60 mg dose to achieve the adequate response to Palynziq.
Shares of BioMarin inched up 2.9% following the aforementioned news on Wednesday. However, the stock has declined 8.1% so far this year compared with no change for the industry.
Per the company, the approval for higher dose also includes the addition of longer-term efficacy data on Palynziq’s label, which demonstrated sustained Phe lowering out to three years and more than six years of data further supporting the safety profile of Palynziq.
PKU is a rare genetic enzyme deficiency disorder that manifests at birth and if left untreated, causes high levels of Phe concentrations, which induce a variety of cumulative toxic effects on the brain. Per the company, approximately 70,000 individuals in various countries are diagnosed with PKU.
We note that Palynziq is the first enzyme therapy approved for the treatment of PKU in the United States and Europe. In the first six months of 2020, Palynziq recorded sales worth $75.3 million, reflecting an increase of 142% year over year. The label expansion to 60 mg dose should help Palynziq treat a broader patient population and drive its sales in the future.
Palynziq is essential for BioMarin’s long-term growth, given its other PKU treatment Kuvan facing a generic threat. Per settlements with Dr Reddy’s RDY and Par Pharmaceuticals, Kuvan generics are expected to enter the United States market beginning this month.
This apart, a third PKU treatment option in BioMarin’s PKU franchise is its gene therapy candidate BMN 307. A phase I/II study on BMN 307 began in September 2020. Earlier this month, the FDA granted a Fast Track designation to BMN 307 for PKU.
Zacks Rank & Stocks to Consider
BioMarin currently carries a Zacks Rank #4 (Sell).
Better-ranked stocks in the healthcare sector include AVEO Pharmaceuticals, Inc. AVEO and Editas Medicine, Inc. EDIT, both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
AVEO’s loss per share estimates have narrowed 25.6% for 2020 and 28.6% for 2021 over the past 60 days. The stock has rallied 7% year to date.
Editas’ loss per share estimates have narrowed 3.6% for 2020 and 0.8% for 2021 over the past 60 days. The stock has increased 4.9% year to date.
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