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BioMarin BMRN has announced positive results from the phase III PEGASUS study evaluating Palynziq (pegvaliase) in adolescents aged 12-17 years with phenylketonuria (PKU).
The study achieved its primary endpoint – patients treated with Palynziq achieved a statistically significant lowering in blood phenylalanine (Phe) levels compared to diet alone. The safety results were also consistent with the known profile of the drug.
Palynziq is already approved in the United States to reduce blood Phe concentration in adults aged 18 years and older with PKU. The drug is currently the only enzyme therapy to treat the disease in this age group.
PKU is a rare genetic disorder that prevents the body from breaking down Phe, an amino acid found in protein-rich foods, leading to toxic levels that can cause severe neurological damage. While newborn screening enables early diagnosis, lifelong dietary management is challenging, creating demand for innovative treatments like those developed by BioMarin.
BMRN’s Future Plans for Palynziq
Based on the encouraging PEGASUS results, BioMarin plans to submit a regulatory filing with the FDA seeking label expansion for the drug in adolescents with PKU.
The company plans to present detailed results from the PEGASUS study at a future medical meeting.
BMRN’s Stock Performance
Shares of BioMarin have outperformed the industry year to date, as seen in the chart below.
Image Source: Zacks Investment Research
Palynziq – A Key Product in BMRN’s Portfolio
Palynziq is one of BioMarin’s most successfully marketed products. The company added $355 million from the PKU drug’s sales, indicating 17% year-over-year growth, driven by strong commercial uptake in the United States.
A potential approval for Palynziq in the adolescent population will likely help boost the drug’s future sales. We believe the drug has a peak commercial opportunity of roughly $1 billion, based on the encouraging Phe reductions achieved in PKU patients in pivotal clinical studies.
BMRN’s Zacks Rank
BioMarin currently carries a Zacks Rank #3 (Hold).
BioMarin Pharmaceutical Inc. Price
BioMarin Pharmaceutical Inc. price | BioMarin Pharmaceutical Inc. Quote
Key Picks Among Biotech Stocks
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