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 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

At #JPM26 in San Francisco, we will discuss the promising outlook of our Skeletal Conditions and Enzyme Therapies business units, provide updates on our pipeline, and offer additional perspective on the integration of Inozyme Pharma and pending acquisition of Amicus Therapeutics. We’re looking forward to connecting with the broader biopharma community and highlighting our impact on rare disease communities around the world. Join our presentation at 11:15 a.m. PT / 2:15 p.m. ET on Monday, Jan. 12 to learn more: ms.spr.ly/6013tf3hN

Today we joined @VeevaSystems in announcing a long-term enterprise agreement that expands our longstanding partnership. This agreement will help us increase our speed, agility and operational efficiency, and optimize the healthcare professional and patient experience. Learn more in our joint press release: ms.spr.ly/6010tN1OE

We’re excited to announce that we have entered into a definitive agreement to acquire Amicus Therapeutics, adding new therapies for Fabry disease and Pompe disease. This transaction has the potential to expand BioMarin’s position as a leader in rare diseases, enabling these innovative medicines to reach more individuals around the world and strengthening our financial outlook. Read more in our press release: ms.spr.ly/6042toNsG $BMRN

Raegan is an energetic 11-year-old living with #achondroplasia, who is at her happiest when spending time with family and being active with friends. That made Camp Ellellbee – an adaptive camp for people living with genetic skeletal conditions – the perfect place for Raegan and her family to have fun together while connecting with others who understand their journey and learning from each other’s lived experiences. Hear from Raegan’s mom, Raechel, to learn more about her family and what made Camp Ellellbee a significant and memorable step in the family’s journey. Learn more: ms.spr.ly/6016trNPE

A study published in Molecular Genetics & Genomic Medicine, co-authored by our colleagues Stacie Cavallaro and Margaret Cho, sheds new light on the experiences of people living with #hypochondroplasia and their families. The study found that many families wait months or see multiple specialists before receiving an accurate diagnosis due to limited familiarity with the condition. It also showed that hypochondroplasia affects more than growth, with many participants reporting learning differences, developmental delays and emotional challenges that shape daily life. Families shared that these challenges often leave them searching for better information, guidance and community support. Thank you to Dr. Andrew Dauber, Chandler Crews and Renata Pekala for your collaboration on this important work. By learning from the community, we can help improve understanding, diagnosis and support for people with rare skeletal conditions. Learn more about the study: bit.ly/4pe6bzk

When Kristen DeAndrade initially imagined what a camp for people living with skeletal conditions and their families might look like, as someone who had little experience with camps, she wasn’t sure how it would all come together. But the Founder and President of the Little Legs Big Heart Foundation had a clear vision for the objectives of bringing the community together in such a setting. “I wanted it to be a home away from home – a place where people could make those connections that truly matter, have the hard conversations, where kids could form bonds with other kids, teens, their camp counselors,” Kristen says of what would become Camp Ellellbee. “And honestly, it has exceeded my expectations.” Held for the first time throughout this fall, Camp Ellellbee has brought together more than 60 families from the U.S. dwarfism community for weekends filled with traditional camp activities, laughter and connections meant to broaden support networks and encourage open dialogue about living with genetic skeletal conditions. Hear from Kristen and others who attended and supported Camp Ellellbee to learn what the experience meant to them. Learn more: bit.ly/4o4mNsu

The @US_FDA has accepted the supplemental Biologics License Application (sBLA) for Priority Review to expand our medicine’s indication to adolescents living with #PKU, a rare, inherited metabolic condition that affects the body’s ability to metabolize a part of a protein called phenylalanine (Phe). Read more in our press release: bit.ly/4nprpcf

Today we announced Q3 2025 results to the investor community, including an 11% year-over-year increase of year-to-date total revenues, as we continue to deliver on our strategy and reach patients around the world. Read more in our press release: bit.ly/48Qev3I $BMRN

We’re looking forward to sharing an update with investors on our Q3 2025 results Monday, Oct. 27 at 4:30 p.m. ET / 1:30 p.m. PT. Join our earnings call and webcast here: bit.ly/4qIZnvx $BMRN