BMRN

EX-99.1

2 earningsrelease-2nov26ex991.htm

EX-99.1

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Exhibit 99.1

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Investors:Media:

Traci McCartyMarni Kottle

BioMarin Pharmaceutical Inc.BioMarin Pharmaceutical Inc.

(415) 455-7558(650) 374-2803

BioMarin Reports Third Quarter 2026 Financial and Operating Results

Third Quarter 2026 Total Revenues Increased 32% Y/Y to $1.025 billion; Growth Driven by 44% Increase in Metabolic Conditions Y/Y, and 12% Increase in Skeletal Conditions Y/Y

Updates Full-year 2026 Guidance to Reflect Order Timing Uncertainty and Impact of Two Acquired In-process Research and Development (IPR&D) Charges

Recently Announced the Addition of a New Investigational Fabry Asset to Clinical Portfolio

PDUFA Action Date of May 30, 2027 for VOXZOGO® for the Treatment of Hypochondroplasia (HCH)

Conference Call and Webcast Scheduled Today at 4:30 p.m. ET

SAN RAFAEL, Calif., November 2, 2026 – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced financial results for the third quarter ended September 30, 2026.

“BioMarin delivered a strong quarter, with revenue growth of 32%, driven by a more diversified portfolio following the acquisition of Amicus Therapeutics earlier this year and increased demand for our medicines from patients around the world. For the first time, quarterly revenue exceeded $1 billion, a milestone for BioMarin,” said Alexander Hardy, President and Chief Executive Officer of BioMarin. “We are building on this achievement by continuing to expand our pipeline through focused business development. We are excited to add our newest asset, an investigational treatment for Fabry disease, which has the potential to become the first subcutaneous enzyme therapy for this condition. With this fourth new asset added to our pipeline in 2026, we are following through on our commitment to build sustainable growth for the future.” Mr. Hardy added, “As we approach 2027, we are well positioned for our next chapter of growth, including the potential launch of VOXZOGO in hypochondroplasia and the continued execution of our financial and innovation strategy.”

1

2026 Business and Pipeline Highlights

Innovation

•BioMarin is accelerating innovation by expanding its pipeline through business development. Four transactions in 2026 have added development programs from preclinical through Phase 3:

◦BMN 450 (HM15421/GC1134A) is an investigational long-acting enzyme replacement therapy (ERT) that has the potential to be the first subcutaneous ERT for Fabry disease. Now in a global Phase 1/2 clinical study, it could extend BioMarin’s reach beyond patients with genetic variants amenable to GALAFOLD®.

◦BMN 440 (ALE1) for hypophosphatasia (HPP) is a potential first-in-class oral therapy for HPP. The Phase 1/2a study in healthy volunteers and adults with HPP is ongoing, with data expected in 2027.

◦BMN 430 for ReNU syndrome has the potential to be a first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies. Preclinical studies are underway to identify a lead ASO candidate.

◦BMN 820 (DMX-200) for focal segmental glomerulosclerosis (FSGS) is a first-in-class oral CCR2 inhibitor for which BioMarin holds exclusive U.S. commercialization rights. The Phase 3 ACTION 3 trial is fully enrolled, with pivotal data expected in 2028.

•Today, BioMarin announced that the U.S. Food and Drug Administration (FDA) has set a PDUFA target action date of May 30, 2027 for its supplemental NDA to expand the use of VOXZOGO for the treatment of HCH, the first potential targeted therapy for the condition. BioMarin also announced today that the European Medicines Agency (EMA) validated its submission for the approval of VOXZOGO to treat HCH.

•BioMarin’s application for full approval of VOXZOGO in children with achondroplasia is under review by the FDA, with a PDUFA target action date of February 28, 2027. The application includes long-term safety and efficacy data, such as final adult height and clinical outcomes beyond linear growth.

•The Phase 2/3 study for BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia, continues to enroll on track. Dose-response data from this study is expected in 2027.

•In October, five-year results from the PROPEL open-label extension study with POMBILITI® + OPFOLDA® were presented in a late-breaking presentation at the World Muscle Society Annual Congress. In adults with late-onset Pompe disease, POMBILITI + OPFOLDA demonstrated durable motor function outcomes and relative stabilization of pulmonary function. This included people who had received enzyme replacement therapy for a mean of 7.6 years before enrolling. No new safety signals were identified.

•For BMN 351, BioMarin’s Phase 1/2 candidate for Duchenne muscular dystrophy, the company expects to provide a program update by year-end.

Growth

•In the first full quarter as part of BioMarin, GALAFOLD and POMBILITI + OPFOLDA delive

bmrn-20261006

0001048477false00010484772026-10-062026-10-06

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 OR 15(d) of The Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): October 6, 2026

BioMarin Pharmaceutical Inc.

(Exact name of registrant as specified in its charter)

Delaware 000-26727 68-0397820

(State or other jurisdiction of incorporation or organization) (Commission File Number) (I.R.S. Employer Identification No.)

770 Lindaro Street

San Rafael

California

94901

(Address of Principal Executive Offices)

(Zip Code)

(415) 506-6700

(Registrant's telephone number, including area code)

Not Applicable

(Former name or former address, if changed since last report.)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each classTrading Symbol(s)Name of each exchange on which registered

Common Stock, par value $0.001BMRNThe Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 2.02 Results of Operations and Financial Condition

Estimated Financial Impact of the Alesta Acquisition

In connection with its acquisition of Alesta Therapeutics B.V. (Alesta), a Dutch private limited liability company (besloten vennootschap met beperkte aansprakelijkheid), which was completed on August 31, 2026, BioMarin Pharmaceutical Inc. (BioMarin) expects its GAAP and non-GAAP financial results for the third quarter of 2026 to include pre-tax charges of approximately $283 million of acquired in-process research and development (IPR&D) expense. The IPR&D charge and the ongoing operating expenses represent an unfavorable impact of approximately $1.50 to both GAAP Diluted earnings per share (EPS) and Non-GAAP Diluted EPS for the third quarter of 2026.

While acquired IPR&D charges may be incurred upon execution of acquisitions, collaborations, licensing agreements, and other business development transactions, BioMarin does not forecast acquired IPR&D charges because the occurrence, magnitude, and timing of these transactions are inherently uncertain. Accordingly, BioMarin’s full-year 2026 guidance previously announced on August 6, 2026, did not reflect the acquisition of Alesta. BioMarin expects the acquisition to result in total pre-tax charges of approximately $320 million for the full year 2026, consisting of the acquired IPR&D charges mentioned above and ongoing operating expenses, including transaction and transition costs. These charges represent an unfavorable impact of approximately $1.55 to both GAAP Diluted EPS and Non-GAAP Diluted EPS for the full year 2026. BioMarin expects to provide updated full-year 2026 guidance in connection with its third quarter 2026 financial results.

BioMarin’s results for the quarter ended September 30, 2026, have not been finalized and are subject to BioMarin’s financial statement closing procedures. There can be no assurance that actual results will not differ from the preliminary estimates described herein. The preliminary, unaudited financial information in this Current Report on Form 8-K (this Report) does not present all information necessary for a complete understanding of BioMarin’s results for the quarter ended September 30, 2026, and should not be viewed as a substitute for full financial statements prepared in accordance with GAAP.

The information in this Report shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the Exchange Act), or otherwise subject to the liabilities under that section or Sections 11 and 12(a)(2) of the Securities Act of 1933, as amended (the Securities Act), nor shall it be deemed incorporated by reference in any filing under the Securities Act or the Exchange Act, whether made before or after the date hereof, regardless of any general incorporation language in such filing.

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