BMRN's PKU Drug Palynziq Meets Goal in Late-Stage Pediatric Study

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.

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

Some better-ranked stocks from the sector are ANI Pharmaceuticals ANIP, CytomX Therapeutics CTMX and 89bio ETNB. While ANIP and CTMX sport a Zacks Rank #1 (Strong Buy) each at present, ETNB carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2025 earnings per share (EPS) have risen from $5.54 to $6.35. EPS estimates for 2026 have increased from $6.75 to $7.21 during the same period. Year to date, shares of ANIP have risen nearly 26%.

ANIP’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 17.32%.

In the past 60 days, estimates for CytomX Therapeutics’ 2025 EPS have improved from a loss of 31 cents to a profit of 25 cents. During the same timeframe, estimates for loss per share for 2026 have narrowed from 65 cents to 31 cents. Year to date, shares of CytomX have lost 41%.

CTMX’s earnings beat estimates in three of the trailing four quarters and missed the mark once, delivering an average surprise of 180.70%.

Estimates for 89bio’s loss per share have narrowed from $2.66 to $1.98 for 2025 in the past 60 days. During the same time frame, estimates for loss per share for 2026 have improved from $3.06 to $2.15. ETNB’s shares have lost 8% year to date.

89bio’s earnings missed estimates in three of the trailing four quarters and beat the mark on one occasion, delivering an average negative surprise of 46.18%.

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