BioMarin Pharmaceutical Inc. Announces Landmark Phase‑3 Success for PALYNZIQ® in Adolescents with Phenylketonuria
BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) reported that its flagship enzyme‑replacement therapy, PALYNZIQ® (pegvaliase‑pqpz), achieved a 49.7 % reduction in mean blood phenylalanine (Phe) levels among adolescents aged 12‑17 in the late‑stage PEGASUS Phase‑3 study. The data were presented at the 15th International Congress of Inborn Errors of Metabolism (ICM) in San Rafael, Calif., on September 6, 2025, and were subsequently released through a press release on the same day.
Key Findings
| Metric | Result | Significance |
|---|---|---|
| Mean Phe reduction | 49.7 % | Surpasses the pre‑defined clinical threshold for therapeutic efficacy in PKU. |
| Safety profile | Comparable to previous adult trials | Demonstrates consistent tolerability across age groups. |
| Regulatory filing timeline | Second half of 2025 | Aligns with BioMarin’s planned submission to the FDA and EMA. |
The PEGASUS study, comprising 118 adolescents, confirmed that PALYNZIQ consistently lowered Phe concentrations to levels that are considered safe for brain development and cognitive function. This outcome marks a critical milestone for BioMarin’s phenylketonuria (PKU) portfolio, which has long been a core focus of its enzyme‑technology platform.
Strategic Implications
Market Leadership in PKU
- BioMarin positions itself as the first to offer a robust, once‑weekly subcutaneous therapy for adolescents—a demographic that previously had limited treatment options.
- The strong efficacy data may accelerate market acceptance and reimbursement discussions in the United States and Europe.
Pipeline Synergy
- PALYNZIQ’s success reinforces BioMarin’s broader enzyme‑replacement strategy, which includes products for lysosomal storage disorders and burn care.
- Positive Phase‑3 data enhance investor confidence in the company’s translational capabilities and can create cross‑product goodwill.
Financial Outlook
- With the company’s market cap at $11.26 bn and a recent closing price of $57.77, the impending launch could generate significant revenue streams, further strengthening the company’s cash and free‑cash‑flow position.
- The firm’s recent earnings call highlighted a 52‑week low of $52.93, suggesting room for upside once commercialization begins.
Forward‑Looking Perspective
BioMarin’s planned submission in the latter half of 2025 is expected to coincide with the U.S. FDA’s priority review pathway for orphan drugs, potentially shortening the approval window. Should the therapy receive clearance, BioMarin could capture a sizeable share of the growing PKU treatment market, estimated at $1–$2 bn globally. Moreover, the data support the company’s broader strategy of targeting genetically defined metabolic disorders, an area where the competitive landscape remains fragmented.
Market Context
The Nasdaq Composite, which stood 21,841.12 on September 5, 2025, experienced a modest gain of 0.61 %—reflecting overall market stability amid a backdrop of encouraging biotech headlines. BioMarin’s robust clinical results stand out in a crowded healthcare sector, potentially translating into a rally for the stock in the coming months.
This article synthesizes publicly available information and reflects current developments in BioMarin Pharmaceutical Inc.’s clinical and commercial trajectory. The data cited are from the company’s own press releases and conference presentations as of September 6, 2025.