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FDA Approves BioMarin’s Gene Therapy for a Genetic Bleeding Disorder

Science | June 29, 2023

Jean-Jacques (JJ) Bienaimé is BioMarin's former Chairman and Chief Executive Officer and a current member of our Board of Directors.

We’re thrilled to share that the U.S. Food and Drug Administration has approved our gene therapy for a genetic bleeding disorder.

Developing the one-time therapy and bringing it over the finish line, first in Europe and now the United States, has been a global effort. Our teams have made unprecedented scientific, medical, manufacturing and regulatory progress in their pursuit of the treatment, partnering with investigators, patients and the broader bleeding disorders community. Thank you and congratulations to everyone in the community, especially the patients and physicians who participated in our clinical trials, for your help in reaching this extraordinary milestone.

With the gene therapy, our purpose at BioMarin assumes new meaning. It’s a historic achievement based on decades of genetic advances by BioMarin’s Worldwide Research and Development teams and scientific colleagues around the world. We are ready to meet demand for the treatment, relying on the company’s dedicated manufacturing facility. Based in Novato, California, the facility is in full compliance with Good Manufacturing Practices for gene therapy technology.

At BioMarin, we will continue to build on our key learnings from the gene therapy and apply them to other investigational programs in our pipeline, all of which have potentially transformative science at their core. As we further explore these candidates for the benefit of people with more common genetic conditions, one aspect of our business will remain constant: The most promising science will always drive our path forward.