An Open Letter to the Pompe and Fabry Communities
March 27, 2026
Dear All,
At the end of 2025, BioMarin announced our plans to acquire Amicus Therapeutics. These kinds of agreements require regulatory review in the U.S. and in other countries where the two companies operate, and these reviews take time. We are very much looking forward to the completion of the process, and we remain on track for Amicus to become part of BioMarin in the second quarter of this year. During this transition, we are continuing to operate as two separate companies, which means as much as we’d like to, we are not yet able to fully engage, set up meetings or introduce ourselves in person.
We know that many of you have questions – so in that spirit, we are writing to offer a few words of introduction and to start a conversation that we are looking forward to building on in the coming weeks, months and years. BioMarin has about 3,000 employees and is based in Marin County, California, just north of San Francisco. We were founded almost 30 years ago and specialize in rare diseases, including lysosomal storage diseases. Our story begins with a dad named Mark Dant, whose 3-year-old son, Ryan, had just been diagnosed with a form of mucopolysaccharidosis (MPS). Mark and his family were told that Ryan wouldn’t live to see his 10th birthday, a fate that the family refused to accept. The Dants started the Ryan Foundation, raising $342 with their first event, a bake sale. The Dants’ fundraising and pursuit of a treatment eventually led them to Emil Kakkis and Liz Neufeld at the University of California, Los Angeles. Within a few years, BioMarin licensed the program from UCLA. In 1998, Ryan became the third person to receive an infusion with the then-experimental medicine, which was approved in 2003. Today, Ryan is in his 30s, married and working in healthcare.
The medicine was the first to treat Ryan’s condition and BioMarin has gone on to bring five other “firsts” to patients with other rare diseases, including additional forms of MPS and CLN2/Batten disease. In total, our portfolio includes eight marketed products, as well as numerous clinical and preclinical programs, all in rare disease.
Over the past several months, we have spent time learning from our colleagues at Amicus and reading about Pompe and Fabry, as we prepare for the acquisition to close. It is a great privilege to work in rare diseases and to get to know the children, parents, siblings and caregivers in this community. Their resilience and tenacity is humbling. We are excited to get to know the Fabry and Pompe communities – and committed to listening and learning about your lived experiences.
More to come – and in the meantime, we invite you to learn more about BioMarin on our website. You can also find us on social media. We’re on LinkedIn, Facebook and Instagram. These channels provide more insight into who we are as a company.
Warmly,
Alexander Hardy
President & CEO
Marni Kottle
Chief Corporate Affairs & Patient Advocacy Officer


