BioMarin shares rise as new VOXZOGO and PALYNZIQ data reinforce market potential

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) has once again demonstrated its leadership in the field of genetic and rare disease treatments, revealing new data from clinical trials and real-world studies of its breakthrough therapies. These findings, presented at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Los Angeles, underscore the safety, efficacy, and transformative potential of VOXZOGO (vosoritide) and PALYNZIQ (pegvaliase-pqpz) in treating two distinct but impactful rare genetic disorders: achondroplasia and phenylketonuria (PKU).

With the rare disease space continuing to draw increasing attention from both pharmaceutical companies and investors, BioMarin‘s latest clinical data has significant implications not only for patient care but also for the market at large. VOXZOGO, which targets achondroplasia, a genetic condition that leads to abnormal bone growth and dwarfism, and PALYNZIQ, which addresses PKU, a metabolic disorder, are both vital therapies that have the potential to reshape long-term treatment paradigms for these debilitating conditions.

This article delves into the latest data shared by BioMarin, its implications for patient outcomes, and its broader impact on the pharmaceutical industry. By providing insights into the safety profiles, efficacy data, and real-world treatment adherence of VOXZOGO and PALYNZIQ, we aim to offer an in-depth analysis of how these therapies are changing the landscape of rare disease treatments.

How Effective Is VOXZOGO in Treating Achondroplasia in Very Young Children?

Achondroplasia is a genetic condition characterized by dwarfism, where bone growth is impaired, particularly in the limbs, leading to a range of physical and developmental challenges. Treatment options for achondroplasia have historically been limited, but VOXZOGO represents the first FDA-approved therapy designed specifically for children with this condition. The latest real-world data presented by BioMarin underscores the high treatment adherence and favorable safety profile of VOXZOGO in very young children—specifically those under the age of three.

The Japanese study showed impressive results from a cohort of 63 children, all receiving VOXZOGO treatment for up to 23.7 months. Importantly, no treatment-related adverse events were reported, and no dose interruptions occurred during the study period. These findings validate the results from previous clinical trials, which demonstrated that VOXZOGO can stimulate growth in children with achondroplasia, addressing some of the physical limitations associated with the condition. The safety profile, which included infants as young as one month, further supports the therapeutic potential of initiating treatment early in life.

As Dr. Greg Friberg, BioMarin’s EVP and Chief R&D Officer, highlighted, early initiation of VOXZOGO in young children can translate into greater benefits over time. Given that the effects of restricted growth due to achondroplasia become apparent from birth, BioMarin’s research suggests that earlier treatment may lead to more significant improvements in both physical development and quality of life. With these findings, BioMarin is set to push forward with further clinical studies, particularly in skeletal conditions like hypochondroplasia, which also affects bone growth but to a lesser extent.

What Does New Data on PALYNZIQ Tell Us About Managing Phenylketonuria (PKU)?

In parallel to VOXZOGO’s success, BioMarin also presented significant findings for PALYNZIQ, an enzyme substitution therapy for phenylketonuria (PKU). PKU is a rare metabolic disorder where the body is unable to process phenylalanine (Phe), an amino acid found in many foods, leading to dangerous levels of Phe in the blood. This condition can cause severe intellectual disability if not properly managed.

The new data, derived from the OPAL study, further reinforces PALYNZIQ’s efficacy and safety in reducing blood Phe levels, offering substantial improvements in the quality of life for adults living with PKU. In this observational trial, baseline blood Phe levels were 1029 μmol/L, which decreased by 67.8% to 293 μmol/L at week 96. This dramatic reduction in blood Phe not only lowers the risk of intellectual and neurological impairments associated with PKU but also correlates with improvements in health-related quality of life (HRQoL).

Secondary data from the Phase 3 PRISM trial also showed that maintaining lower blood Phe levels was linked to cognitive and emotional improvements in patients. Specifically, participants with sustained Phe levels of ≤120 μmol/L had significantly better scores in attention and mood compared to those with higher Phe levels. These findings suggest that long-term metabolic control through PALYNZIQ not only reduces the risk of physical complications but also offers substantial neuropsychological benefits for patients with PKU.

BioMarin’s ATLAS study provides further insight into how PALYNZIQ is reshaping the treatment landscape for PKU. Data from 19 U.S. clinics indicate that an increasing number of patients are able to achieve blood Phe levels ≤360 μmol/L, while fewer individuals experience dangerously high Phe levels (>1200 μmol/L), which is a critical milestone in PKU management.

What Are the Financial Implications of BioMarin’s Latest Clinical Data?

The positive clinical outcomes for both VOXZOGO and PALYNZIQ present significant opportunities for BioMarin to not only strengthen its market position in rare disease therapeutics but also to deliver substantial financial returns for its investors. The company has seen a decline in its stock price over the past year, with shares trading at approximately $71.39 as of March 20, 2025, down around 16.86% from the previous year. However, analyst sentiment remains largely favorable, with a Buy rating consensus and a 12-month price target of $97.73, indicating a potential upside of over 35%. This suggests that the market is optimistic about BioMarin’s future prospects, especially given the success of its therapies for achondroplasia and PKU.

In terms of financial projections, analysts anticipate that BioMarin’s earnings per share (EPS) for 2025 will rise to $3.17, compared to $2.26 in 2024. Furthermore, the company’s revenue is expected to increase to $3.20 billion, a growth of over 12% from the previous year, driven by the continued adoption of VOXZOGO and PALYNZIQ in clinical and real-world settings. These financial projections reflect the significant commercial potential of BioMarin’s therapies as more data supports their effectiveness.

For investors, the growing real-world evidence supporting the safety and efficacy of BioMarin’s therapies strengthens the argument for continued investment in the company, with the possibility of market upside once the company secures additional regulatory approvals and further clinical trial results.