BMRN — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-05

• (1) In 2026, we announced that we will no longer market ROCTAVIAN. For additional information related to ROCTAVIAN, see Note 19 to the Consolidated Financial Statements accompanying our Annual Report on Form 10-K for the year ended December 31, 2025.

• Management’s Discussion and Analysis of Financial Condition and Results of Operations (continued)

• •In April 2026, we completed the acquisition of Amicus Therapeutics, Inc. (Amicus), a publicly traded, global, biotechnology company for $14.50 per share in an all-cash transaction for a total equity value of approximately $4.8 billion. In connection with the acquisition, the Company also repaid in …

• •In April 2026, in connection with the Amicus acquisition, we obtained senior secured term loan facilities for $2.8 billion in aggregate principal and a new $600.0 million senior secured revolving credit facility. Upon entry into the senior secured term loan facilities, the remaining bridge commitme…

• •In April 2026, the first patient was enrolled in the registration-enabling Phase 2/3 study of BMN 333, our long-acting C-type natriuretic peptide (CNP) for achondroplasia.

Text removed vs the prior filing · source: 10-Q · 2025-10-28

• Based on such strategic portfolio assessments, certain programs that do not meet its threshold for further development and commercialization could be discontinued.

• In October 2025, we announced our plan to pursue options to divest ROCTAVIAN, including exploring out-licensing opportunities. We plan to continue to make ROCTAVIAN commercially available in the U.S., Italy and Germany until next steps are finalized. We will continue to provide support and monitorin…

• In September 2025, at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting, we shared new data demonstrating improved spinal morphology – one of the factors that contributes to spinal stenosis, a leading cause of morbidity in achondroplasia – following treatment with VOXZOGO in …

• In September 2025, we presented new data from the PALYNZIQ Phase 3 PEGASUS study in 12 to 17 year-olds demonstrating statistically significant blood phenylalanine (Phe) lowering compared to diet alone.

• In August 2025, we announced that Phase 1 data in its healthy volunteers study with BMN 333, BioMarin’s long-acting C-type natriuretic peptide (CNP), demonstrated area-under-the-curve (AUC) pharmacokinetic (PK) levels greater than three times the levels observed in other long-acting CNP studies, and…

Text added vs the prior filing · source: 10-Q · 2026-05-05

• Our competitors may develop, manufacture and market products that are more effective or less expensive than ours. They may also obtain regulatory approvals for their products faster than we can obtain them (including those products with orphan drug designation, which may prevent us from marketing ou…

• For example, we faced significant uncertainty as to whether gene therapy would gain the acceptance of the public or the medical community. In October 2025, we announced our plan to pursue options to divest ROCTAVIAN, including exploring out-licensing opportunities. Subsequently in December 2025, we …

• *We may not realize the anticipated benefits from the Amicus Acquisition or accurately forecast the future performance of the combined company.

• The success of the Amicus Acquisition will depend, in part, on our ability to realize the anticipated benefits from successfully combining our and Amicus’ businesses. We plan on devoting substantial management attention and resources to integrating our and Amicus’ businesses so that we can fully rea…

• integration process may be disruptive to our and Amicus’ businesses, and, if implemented ineffectively, could restrict realization of the expected benefits of the Amicus Acquisition. In addition, the acquired Amicus business, including GALAFOLD and POMBILITI + OPFOLDA, may not be successful, may req…

Text removed vs the prior filing · source: 10-Q · 2025-10-28

• Our competitors may develop, manufacture and market products that are more effective or less expensive than ours. They may also obtain regulatory approvals for their products faster than we can obtain them (including those products with orphan drug designation, which may prevent us from marketing ou…

• that could block approval) or commercialize their products before we do. With respect to VOXZOGO, other companies are developing, and may in the future develop, products for treatment for achondroplasia that, if approved, could potentially compete with VOXZOGO even during the period of orphan drug e…

• We also face uncertainty as to whether gene therapy will gain the acceptance of the public or the medical community. The commercial success of ROCTAVIAN will depend, in part, on the acceptance of physicians, patients and third-party payers of gene therapy products in general, and our product in part…

• Activist investor actions threatened or commenced against us have and could in the future cause us to incur substantial costs, divert management's attention and resources, cause uncertainty about the strategic direction of our business and adversely affect our business, financial position and result…

• We have been, and may in the future be, subject to activities initiated by activist investors. For example, in December 2023, we entered into a Cooperation Agreement with Elliott Investment Management L.P., Elliott Associates, L.P. and Elliott International, L.P., which expired in December 2024 purs…

Text added vs the prior filing · source: 10-Q · 2026-05-05

• During the three months ended March 31, 2026, none of our directors and officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted or terminated any “Rule 10b5-1 trading arrangement” or “non-Rule 10b5-1 trading arrangement,” as those terms are defined in Item 408 of Regulation S-K.

Text removed vs the prior filing · source: 10-Q · 2025-10-28

• During the three months ended September 30, 2025, none of our directors and officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted or terminated the contracts, instructions or written plans for the purchase or sale of BioMarin securities.