Provides New Corporate Strategy and Introduces 2027 Financial Guidance

BioMarin is an international biotech firm with a portfolio of eight products designed to address life-threatening diseases. Since our inception in 1997, we have leveraged our deep scientific knowledge of genetic disorders to develop breakthrough therapies, utilizing various treatment approaches. Our approved drugs provide solutions for conditions such as achondroplasia, severe hemophilia A, and several rare genetic and lysosomal storage disorders. Additionally, we are progressing a robust pipeline of experimental medicines, currently undergoing both clinical trials and preclinical research.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) held an Investor Day on Sept. 4, 2024, in New York, where President and CEO Alexander Hardy, alongside other executives, outlined the company’s fresh corporate strategy focused on delivering sustainable value and introduced long-term projections for total revenue and Non-GAAP operating margins. The presentation and webcast replay are accessible at investors.biomarin.com.

“In the past nine months, we have worked diligently to transform BioMarin’s operations and processes with the goal of speeding up growth and generating significant value for our stakeholders—patients, employees, and investors,” said Hardy. “Our updated strategy leverages BioMarin’s unique strengths in creating and leading new therapeutic markets globally, allowing us to bring innovative treatments to the patients who need them. We are confident in achieving the ambitious objectives presented today and excited to implement our new vision for BioMarin’s future.”

**Corporate Strategy**

BioMarin’s leadership highlighted the company’s distinct approach, featuring a new organizational structure composed of three business units: Enzyme Therapies, Skeletal Conditions, and ROCTAVIAN®. These units are designed to focus on sustainable growth. Additionally, the strategy includes a $500 million cost transformation initiative, which is expected to help meet Non-GAAP operating margin targets starting in 2026.

**Value Commitment**

The company also presented its long-term financial forecast, which includes plans for revenue growth and expanded Non-GAAP operating margins. The following table outlines BioMarin’s reaffirmed full-year 2024 guidance, as well as the newly introduced financial targets for 2026 and additional metrics for 2027, shared at the Investor Day event.

**Innovation**
BioMarin outlined its innovation strategy, which aims to support the company’s robust pipeline of high-impact treatments. This includes the planned launch of 11 products by 2034, with two expected by 2027. Leveraging its 25-year history of developing treatments for serious genetic disorders, several programs are progressing through its pipeline, including:

– **VOXZOGO®**: Phase 3 trials for treating hypochondroplasia, with data expected in 2026 and potential approval in 2027.
– Clinical updates on four skeletal conditions—idiopathic short stature, Noonan Syndrome, Turner Syndrome, and SHOX Deficiency—currently in or entering Phase 2 trials.
– **PALYNZIQ®**: Label expansion to cover ages 12-17 in the U.S. and 12-15 internationally, with filings planned for late 2025 and early 2026, respectively.
– **BMN 390**: A treatment for phenylketonuria (PKU), designed to reduce immunogenicity through novel pegylation, with an Investigational New Drug (IND) application expected in late 2025.
– **BMN 351**: A therapy for Duchenne Muscular Dystrophy (DMD) targeting a novel exon-skipping site, potentially enabling up to 40% dystrophin expression based on preclinical findings. Proof of Concept (POC) is anticipated in 2025.
– **BMN 349**: A treatment for Alpha-1 Antitrypsin Deficiency with a unique mechanism that could improve liver function. Targeted POC is expected in 2026.
– **BMN 370**: Designed to treat von Willebrand Disease (vWD) by normalizing bleeding events through a single subcutaneous injection. IND submission is expected by late 2025.

**Growth**
BioMarin emphasized its strategy to strengthen its Enzyme Therapies unit while establishing long-term leadership across multiple skeletal conditions, driven by VOXZOGO’s success in treating achondroplasia. Together, these business units are projected to deliver a mid-teen Compounded Annual Growth Rate (CAGR) through 2034.

A replay of the presentation and related slides can be found at investors.biomarin.com.

**About BioMarin**
Established in 1997, BioMarin is a global biotechnology company committed to transforming lives through genetic discovery. The company develops and commercializes therapies that directly target the root causes of genetic disorders. With extensive R&D capabilities, BioMarin has introduced eight transformative treatments for rare genetic diseases. The company’s distinct approach has led to a diverse portfolio of commercial, clinical, and preclinical candidates, addressing significant unmet needs with opportunities for first-to-market or superior benefits over current treatments. For more information, visit www.biomarin.com.

**Forward-Looking Statements**
This release and the associated conference call/webcast contain forward-looking statements about BioMarin’s business, including expectations regarding financial performance, future revenues, Non-GAAP metrics, operating margins, and strategic initiatives. These forward-looking statements also include projections about the clinical development, regulatory approval, and commercialization of BioMarin’s product candidates, such as VOXZOGO, PALYNZIQ, BMN 390, BMN 351, BMN 349, BMN 370, and others. The statements reflect BioMarin’s current beliefs and are subject to risks and uncertainties that could cause actual results to differ. Factors impacting these forward-looking statements are detailed in the company’s filings with the U.S. Securities and Exchange Commission (SEC), particularly the “Risk Factors” section of its quarterly report on Form 10-Q for the quarter ending June 30, 2024.

BioMarin does not commit to updating these statements, except as required by law. Please consider all risks before making investment decisions. Do not rely unduly on forward-looking statements as they reflect only management’s views as of the date made.

**Trademarks**
BioMarin®, BRINEURA®, KUVAN®, NAGLAZYME®, PALYNZIQ®, ROCTAVIAN®, VIMIZIM®, and VOXZOGO® are registered trademarks of BioMarin or its affiliates. ALDURAZYME® is a registered trademark of BioMarin/Genzyme LLC. All other trademarks belong to their respective owners.

**Non-GAAP Information**
This press release contains Non-GAAP financial measures, including Non-GAAP Income, Operating Margin, and Diluted EPS. These measures exclude certain items like amortization, stock-based compensation, and other specified expenses. BioMarin uses both GAAP and Non-GAAP data to assess performance, guide business decisions, and compare results across periods. Investors should consider Non-GAAP measures alongside GAAP information for a comprehensive understanding of BioMarin’s financial health.

**Forward-Looking Non-GAAP Financial Measures**
BioMarin does not provide GAAP guidance (other than revenue) or reconciliation for forward-looking Non-GAAP measures due to the unpredictability of factors like strategic reviews, asset impairments, or investment fluctuations. These items are uncertain and could materially affect GAAP outcomes.

For more detailed financial information, refer to BioMarin’s filings with the SEC.