#United States #San Rafael #BioMarin Pharmaceutical #VOXZOGO #PALYNZIQ

Financial Performance Overview

On October 27, 2025, BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) provided an update on its financial results for the third quarter ended September 30, 2025. The company reported total revenues of $776 million, marking a 4% increase compared to $746 million in the same quarter of the previous year. The growth is primarily attributed to significant revenue increases from its key products, VOXZOGO and PALYNZIQ, both showing more than 20% growth year-over-year.

According to BioMarin's President and CEO, Alexander Hardy, the contributions from their Enzyme Therapies and Skeletal Conditions business units have exceeded expectations. The result underscores the company’s strategic focus on these areas, generating revenue and driving innovative therapies compliant with the needs of patients and stakeholders.

Revenue Details

The strong performance in Q3 is notable, particularly in how each segment has contributed:

• - VOXZOGO: Revenue was $218 million, a 15% increase compared to Q3 2024.
• - PALYNZIQ: Also saw robust growth, amounting to $109 million in revenue, a 20% increase year over year.

Notably, these figures were partially balanced by lower sales in ALDURAZYME and NAGLAZYME, influenced by timing and order fulfillment issues. The company experienced a GAAP net loss of $31 million in Q3 2025, which contrasts sharply with a net income of $106 million in Q3 2024. It's essential to note that this loss primarily resulted from a $221 million charge related to In-Process Research and Development (IPRD) tied to the acquisition of Inozyme Pharma, Inc.

Strategic Revisions

Looking ahead, BioMarin is making strategic decisions, notably moving to divest their product ROCTAVIAN from its portfolio, which highlights their commitment to directing resources towards their high-performing therapies. BioMarin considers ROCTAVIAN valuable for hemophilia A therapy but seeks to ensure patient access through out-licensing arrangements.

The company reaffirmed their full-year revenue guidance, indicating confidence in reaching a range between $3.15 billion to $3.2 billion, capitalizing on the upward trajectory already established in 2025.

Innovations and Future Plans

BioMarin remains dedicated to expansion in therapeutic areas. Key points include:

• - Skeletal Conditions: At the American Society for Bone and Mineral Research conference, they presented promising data related to VOXZOGO's impact on spinal morphology, an aspect crucial for treating achondroplasia.
• - Moving forward, the company plans to initiate a Phase 2/3 study for BMN 333, targeting children with achondroplasia, and anticipates pivotal data for VOXZOGO in treating hypochondroplasia in mid-2026.
• - New indications for VOXZOGO are currently in development, focusing on severe cases and conditions such as idiopathic short stature and Turner syndrome.

Encouraging Market Outlooks

As of Q3 2025, BioMarin has expanded its treatment offerings to children with achondroplasia across 55 countries, with aspirations to reach over 60 countries by 2027. The year-to-date revenue from VOXZOGO soared by 24%, demonstrating the strong market demand. The company sees potential for VOXZOGO's revenue to peak in the final quarter of 2025.

Additionally, PALYNZIQ continues to experience steady growth, achieving a 20% year-over-year increase in total enzyme therapies revenue. This progression highlights BioMarin’s effective strategies in patient treatment, leading to greater adherence and market penetration.

Financial Health

The company's commitment to innovation and operational efficiencies is evident, with operating cash flows totaling $369 million for Q3 and $728 million year-to-date. This financial health supports BioMarin's intent to reinvest in R&D and future growth projects, positioning itself firmly for sustainable development in the biopharmaceutical space.

In conclusion, BioMarin Pharmaceutical is on an upward trajectory, emphasizing specific therapeutic areas while ensuring financial strategies effectively meet business goals. The ongoing commitment to enhancing treatments and expanding market reach reflects a larger vision to improve patient lives affected by rare genetic conditions. Stakeholders and investors can keep their eyes on BioMarin as the company charts its course for innovation and growth in the coming years.