On February 27, 2026, the U.S. Food and Drug Administration approved BioMarin’s PALYNZIQ® (pegvaliase‑pqpz) for use in adolescents 12 years of age and older with phenylketonuria (PKU). The approval expands the drug’s label beyond the adult population, allowing the company to treat a younger cohort that previously had no approved enzyme therapy.
The decision was based on data from the PEGASUS Phase 3 study, which showed a significant mean reduction in blood phenylalanine levels at week 72 compared with diet alone. The study also demonstrated that adolescents could maintain an unrestricted diet while achieving target phenylalanine concentrations, addressing a major clinical challenge in PKU management.
BioMarin’s recent financial results underscore the commercial importance of this approval. Full‑year 2025 revenue rose 13% year‑over‑year to $3.2 billion, driven by 9% growth in Enzyme Therapies and 26% growth in VOXZOGO. PALYNZIQ sales increased 22% year‑over‑year, marking the fourth consecutive quarter of 20% or higher growth. In the fourth quarter of 2025, total revenue grew 17% to $875 million, but GAAP net loss widened to $47 million due to a $240 million charge related to the voluntary withdrawal of ROCTAVIAN. Non‑GAAP income fell to $89 million from $180 million in the prior year. Management guided 2026 total revenue to $3.325 billion–$3.425 billion, excluding the Amicus acquisition, reflecting confidence in continued growth.
Management highlighted the strategic significance of the adolescent approval. President and CEO Alexander Hardy noted that the company’s “operational excellence” underpinned strong 2025 results and that the new indication would “build on this success” in 2026. Executive Vice President and Chief Research & Development Officer Greg Friberg emphasized that expanding PALYNZIQ to adolescents “will allow even more people with PKU the prospect of achieving substantially lower Phe levels.” A clinician from Boston Children’s Hospital added that the approval “provides a genotype‑independent medication that may bring Phe into the normal range while allowing an unrestricted diet.”
The approval was met with a muted market reaction, as investors weighed the Q4 2025 earnings miss on earnings per share and the Roctavian withdrawal. While the announcement was a positive clinical milestone, it was seen as a long‑term growth driver rather than an immediate catalyst for a sharp price move.
Strategically, the adolescent approval fits into BioMarin’s broader rare‑disease portfolio expansion. The company’s pending acquisition of Amicus Therapeutics, expected to close in Q2 2026, will add Galafold and Pombiliti to its pipeline, further diversifying revenue streams. Together, the new indication and the acquisition position BioMarin to capture a larger share of the rare‑disease market and accelerate its growth trajectory.
The content on EveryTicker is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.