BioMarin

At BioMarin, we are driven by our purpose: to be the biotech leader that translates the promise of genetic discovery into medicines that make a profound impact on the life of each patient. The BioMarin 8 – our core Operating Principles – provide the framework for how we work together every day to fulfill that purpose.

Today we announced positive topline results from a Phase 3 pivotal study in children with hypochondroplasia, a genetic skeletal condition for which there are currently no approved treatments. The results mark an important step toward addressing the unmet needs of children living with hypochondroplasia. Learn more: ms.spr.ly/6017vp0dh

Today we provided an update on the Phase 3 ENERGY 3 study evaluating BMN 401 in ENPP1 deficiency. Read more: ms.spr.ly/6019vTKaB

For decades, the National MPS Society has brought families together through conferences, support programs and advocacy initiatives. But as leaders at the organization listened more closely to the community they serve, one reality became increasingly clear in recent years: many families impacted by #mucopolysaccharidosis (#MPS) and #mucolipidosis (#ML) were still not being reached. “We’ve always known the importance of getting families and people together,” says Terri Klein, President and CEO of the National @MPSSociety. “But what if we could bring families together that we didn’t already know – families who may not be members of the Society, who may not be connected in other ways, and who may have more need?” A new approach to community outreach called Crossing Paths emerged as a response to help proactively find families, listen to their realities, and begin breaking down barriers to care and connection. Rather than waiting for families to find the organization, Crossing Paths brings the National MPS Society into neighborhoods and communities across the country where engagement has historically been the lowest, creating welcoming, local gatherings designed to foster trust and understanding. More than three years in, the team has held 18 Crossing Paths events across the country that have helped bring additional families into the fold, driving greater equity and contributing to a more holistic understanding about the needs of the community. “Our biggest learning from Crossing Paths has been that we don’t know what we don’t know. And so we have to listen to the community,” says Leslie Urdaneta, Director of Family Support and Pathways at the National MPS Society. “We have to listen to these families first.” Learn more about Crossing Paths in the second installment of our two-part video series on the National MPS Society’s community programming: ms.spr.ly/6018vuoJ0

When DeAnna and Alex learned that their daughter Savannah was living with #mucopolysaccharidosis type VI (MPS VI), just weeks before her third birthday, the diagnosis landed with a mix of fear, uncertainty and isolation. “We weren’t really sure what to expect,” DeAnna recalls. “It was depressing, for lack of a better word. It was deflating. You felt very alone.” Like many facing a rare condition, DeAnna and Alex were suddenly navigating unfamiliar medical terms, unanswered questions and a future that looked very different from what they had imagined. Hope only began to emerge when they contacted the National MPS Society, a patient advocacy organization serving families impacted by #MPS and #mucolipidosis (ML) across the United States. The family was among the first to receive support through the organization’s Pathways Program, an innovative initiative designed to meet families where they are in the earliest – and often most overwhelming – days following diagnosis. Launched in 2018, Pathways provides free face-to-face guidance from trained professionals for the first year following diagnosis. The program offers education, emotional support and practical resources, while linking families and individuals to a broader community of people who understand the realities of living with MPS or #ML. Learn more about Pathways in the first edition of our two-part video series highlighting the @MPSSociety's innovative community programming: ms.spr.ly/6018vMjQy

Today we announced Q1 2026 results to the investor community and increased full-year 2026 total revenue guidance to reflect an accelerated growth rate following BioMarin’s acquisition of @amicusrx1. Read more in our press release: ms.spr.ly/6013vKCoD $BMRN

At #PES2026, we’re highlighting new data in #achondroplasia and #hypochondroplasia as part of our continued efforts to advance care for children with skeletal conditions. #PediatricEndocrinology Read more in our press release: ms.spr.ly/6018vJ4Hr

We’re looking forward to sharing an update with investors on our Q1 results Monday, May 4 at 4:30 p.m. ET / 1:30 p.m. PT. Join our earnings call and webcast here: ms.spr.ly/6010vJO6q

We’re pleased to announce that @amicusrx1 is now part of BioMarin. We look forward to continuing to make a profound impact for rare disease communities around the world, including people living with Fabry disease and Pompe disease. Read more: ms.spr.ly/6013vGu0R

A recent analysis published in @GIMJournal explores the effects of treatment on the health impacts of #achondroplasia in patients across six clinical trials. We’re committed to advancing research in achondroplasia so healthcare providers have more data to ensure children with skeletal conditions receive the best possible care. Learn more: ms.spr.ly/6018vENrC

We announced today that our Board of Directors has appointed Ian Clark as Chair of the Board, subject to his election at our Annual Meeting of Stockholders on June 2. He will succeed Richard (Randy) Meier, who is retiring after two decades on the Board. We thank Randy for his many contributions and years of service and look forward to Ian’s leadership in this role. Read more in our press release: ms.spr.ly/6018v82oQ

Today we provided an update on our clinical programs for Turner syndrome, SHOX deficiency and ACAN deficiency. Read more: ms.spr.ly/6015QWmlX

We announced data in #achondroplasia today at #ACMGtg26, building on the breadth of our research in #achondroplasia and highlighting the importance of early intervention. Learn more about these new findings here: ms.spr.ly/6014QY7HM

We’re looking forward to sharing new research findings across 10 posters and connecting with clinicians, caregivers and advocates at the American College of Medical Genetics and Genomics (ACMG) Annual Meeting. If you’re attending #ACMGMtg26, make sure to stop by and see our team at booth 1807. We’re also excited to share that preregistration for Camp Ellellbee – an adaptive camp offered by the Little Legs Big Heart Foundation, and supported by BioMarin – is kicking off during the meeting. More than a weekend retreat, Camp Ellellbee serves as a platform for fostering community, making space for facilitating meaningful relationships and providing support to families navigating genetic skeletal conditions. Meeting attendees will have the opportunity to learn more and secure their spots in person at the Little Legs Big Heart Foundation booth, and all others can learn more about Camp Ellellbee and preregister here: ms.spr.ly/6018Qcvtw

The U.S. FDA has approved the supplemental Biologics License Application (sBLA) to expand the approved use of our phenylketonuria (PKU) medicine to include adolescents 12 years of age and older with #PKU. This approval is based on data from PEGASUS, a Phase 3 study in adolescents with PKU. Read more in our press release: ms.spr.ly/6011QjPeD

Today we provided an update on our medicine for the treatment of adults with severe hemophilia A. Learn more: ms.spr.ly/6015Qkfe5

Today we announced Q4 and full-year 2025 results to the investor community, including year-over-year total revenue increases of 17% and 13% for the fourth quarter and full year, respectively. Read more in our press release: ms.spr.ly/6016Qk408 $BMRN

We’re looking forward to sharing an update with investors on our Q4 and full-year 2025 results Monday, Feb. 23 at 4:30 p.m. ET / 1:30 p.m. PT. Join our earnings call and webcast here: ms.spr.ly/6018QX6bQ

We’re excited to join @UCBerkeley in announcing a new research collaboration with the university’s @MTI_UCB to help accelerate the discovery and development of innovative therapies for rare genetic conditions. The initial phase of the collaboration will support specific joint research projects between BioMarin scientists and UC Berkeley researchers. The partnership will bring together the university’s strengths in fundamental disease biology, novel therapeutic technologies and next-generation drug discovery with BioMarin’s deep expertise in translating science into transformational medicines. Read more: ms.spr.ly/6015QnuJ3

A BioMarin study recently published in the American Journal of Human Genetics adds to a growing body of evidence supporting the therapeutic rationale for targeting natriuretic peptide receptor 2 (NPR2), the receptor for C-type natriuretic peptide (CNP), as a way to promote growth in children with genetic skeletal conditions. Our scientists are committed to continuing to study how modulating this pathway can impact the health of people living with these conditions. Learn more: ms.spr.ly/6015QGWFU

We’re thrilled to welcome Arpit Davé to BioMarin as Executive Vice President, Chief Digital and Information Officer. Arpit brings more than 20 years of experience in IT and AI in the biopharmaceutical industry, with a deep track record of leveraging technology to deliver for patients and the business. As a member of the BioMarin Leadership Team, Arpit will reimagine and execute our enterprise technology strategy, data science and digital transformation to help create value for patients, employees and shareholders. Learn more: ms.spr.ly/6014tf4fg