BioMarin’s PALace Study: Long-Term Safety Check on Palynziq and What It Means for Investors

Biomarin Pharmaceutical Inc. (BMRN) announced an update on their ongoing clinical study.

Study Overview

BioMarin Pharmaceutical Inc. is running a long-term safety study called “A Multi-Center, Observational Study to Evaluate the Long-Term Safety of Subcutaneous Injections of Palynziq® (Pegvaliase) in Subjects With Phenylketonuria.” The study, also known as PALace, tracks patients with phenylketonuria (PKU) who are using Palynziq in routine care. Its main goal is to better understand the long-term safety profile of the drug, including allergic reactions and overall tolerability over 10 years, as well as how well added safety measures work in the EU. For investors, this type of post-marketing study is important because it can confirm or challenge the risk profile of an already approved product, affecting long-term revenue and regulatory confidence.

Intervention/Treatment

The treatment being assessed is Pegvaliase, sold as Palynziq, a drug given by subcutaneous injection. It is used to reduce high phenylalanine levels in adults with PKU, a rare metabolic disorder. In this study, Pegvaliase is not assigned by the trial team; instead, patients are observed while receiving the drug as part of their usual care. The focus is on capturing real-world safety outcomes rather than testing different doses or new regimens.

Study Design

The PALace study is observational, not a traditional randomized trial. There is a single cohort of patients who are either starting Pegvaliase within 30 days of joining or already on treatment at enrollment. There is no random assignment, no control group, and no blinding. Researchers simply follow these patients over time and record safety events and outcomes. The primary purpose is safety monitoring in real-world use, which helps regulators, clinicians, and investors understand how the product behaves outside tightly controlled trial settings.

Study Timeline

The study was first submitted to the registry on April 3, 2023, marking the formal start of its public record and signaling BioMarin’s commitment to long-term follow-up. The trial is designed as a 10-year effort, so primary and final completion dates extend well into the 2030s, reflecting the need for prolonged safety tracking in chronic therapy. The latest update to the listing was submitted on May 10, 2024, and the current status is “recruiting,” meaning new patients are still being added. These dates matter for investors because they frame when more meaningful interim safety trends could emerge and how long Palynziq’s post-marketing risk profile will remain under close scrutiny.

Market Implications

From a market standpoint, this update reinforces that BioMarin is actively managing long-term safety for a key rare-disease asset. As a post-marketing, non-interventional study, PALace is more about risk control than near-term upside; it is unlikely to trigger rapid stock moves on its own, but steady, clean safety data over time would support the durability of Palynziq revenue and strengthen BioMarin’s reputation with regulators. Any signal of serious safety concerns could pressure the stock and invite tighter labeling or usage limits, while reassuring findings would help maintain physician confidence, especially as genetic therapies and other metabolic treatments advance. In the broader rare-disease and metabolic space, competitors in PKU and related indications are watching how established therapies like Palynziq fare in long-term real-world use, and investors will factor this into expectations for BioMarin’s competitive edge and lifecycle management.

The PALace study is ongoing and was recently updated, with further details available on the ClinicalTrials portal.

To learn more about BMRN’s potential, visit the Biomarin Pharmaceutical Inc. drug pipeline page.