BMRN — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-11-03

• (In millions of U.S. dollars, except as otherwise disclosed)

• During 2026, we completed the acquisitions of Amicus Therapeutics, Inc. (Amicus) and Alesta Therapeutics B.V. (Alesta). The accounting impacts of these transactions have been reflected in the Condensed Consolidated Financial Statements. Refer to Note 2 – Acquisitions for additional details related t…

• (In millions of U.S. dollars, except as otherwise disclosed)

• •In November 2026, we announced that the U.S. Food and Drug Administration (FDA) set a Prescription Drug User Fee Act (PDUFA) target action date of May 30, 2027 for supplemental new drug application (sNDA) to expand the use of VOXZOGO for the treatment of hypochondroplasia (HCH). We also announced t…

• •In October 2026, we entered into an exclusive worldwide license agreement with GC Biopharma Corp. (GC Biopharma) and Hanmi Pharm Co., Ltd. (Hanmi) to develop and commercialize BMN 450 (also known as HM15421/GC1134A), an investigational long-acting enzyme replacement therapy for Fabry disease. Under…

Text removed vs the prior filing · source: 10-Q · 2026-08-06

• On April 27, 2026, we completed the acquisition of Amicus Therapeutics, Inc. (Amicus). The results of operations of Amicus, along with the preliminary estimated fair values of assets acquired and liabilities assumed in the acquisition, have been included in the Condensed Consolidated Financial State…

• •In July 2026, we submitted our supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) for the approval of VOXZOGO for the treatment of hypochondroplasia following our announcement in May 2026 that the Phase 3 CANOPY-HCH-3 study met its primary endpoint.

• •In July 2026, we announced that the FDA accepted our sNDA for full approval of VOXZOGO in children with achondroplasia, with a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2027.

• •In July 2026, BioMarin and the n-Lorem Foundation entered into a collaboration and global exclusive license agreement to develop a potential first-in-disease antisense oligonucleotide (ASO) medicine for ReNU syndrome, a serious, rare neurodevelopmental condition with no approved targeted therapies.

• •In June 2026, the European Commission approved PALYNZIQ for adolescents 12 years and older with PKU. In February 2026, FDA approved PALYNZIQ for adolescents 12 years of age and older with PKU.

Text added vs the prior filing · source: 10-Q · 2026-11-03

• potential market for and profitability of a drug. We cannot predict whether the FDA will request, seek to require or ultimately require modifications to, or impose additional requirements on, the PALYNZIQ REMS program, or whether the FDA will permit modifications to the PALYNZIQ REMS program that we…

• Several healthcare reform initiatives culminated in the enactment of the Inflation Reduction Act (IRA) in August 2022, which, among other things, eliminated, beginning in 2025, the coverage gap under Medicare Part D by significantly lowering the enrollee maximum out-of-pocket costs and requiring man…

• containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize our products.

• government shutdown occurs, it could significantly impact the ability of the FDA to timely review and process our regulatory submissions, which could have a material adverse effect on our business.

• enforcing patents relating to our products and product candidates, including without limitation composition-of-matter patents, which are generally believed to offer the strongest patent protection.

Text removed vs the prior filing · source: 10-Q · 2026-08-06

• could make it more difficult or expensive for us to distribute PALYNZIQ in the U.S., impair the safety profile of PALYNZIQ, disrupt continuity of care for PALYNZIQ patients and/or negatively affect sales of PALYNZIQ.

• Several healthcare reform initiatives culminated in the enactment of the Inflation Reduction Act (IRA) in August 2022, which, among other things, eliminated, beginning in 2025, the coverage gap under Medicare Part D by significantly lowering the

• enrollee maximum out-of-pocket costs and requiring manufacturers to subsidize, through a newly established manufacturer discount program, 10% of Part D enrollees’ prescription costs for brand drugs below the out-of-pocket limit, and 20% once the out-of-pocket limit has been reached. The IRA also req…

• In addition, we have pursued, and may in the future pursue, litigation, administrative challenges or other types of proceedings to protect our patents and intellectual property rights. Such proceedings are often protracted and expensive, and have unpredictable outcomes, which can include the initiat…

• If we are unable to protect our intellectual property, third parties could develop competing products, which could adversely affect our revenues and financial results generally.

Text removed vs the prior filing · source: 10-Q · 2026-08-06

• On April 27, 2026, we committed to a restructuring plan related to the integration of Amicus. This restructuring plan is intended to realize certain targeted synergies following the acquisition, primarily through reductions in overlapping personnel costs within the general and administrative functio…