A MESSAGE FROM THE CEO
Welcome! Thank you for visiting the BioMarin Pharmaceutical website.
At BioMarin, our strategy is to develop first-to-market or best-in-class therapies which make big differences in the lives of the small number of patients suffering from serious or rare orphan diseases. That strategy bore fruit in 2012, and BioMarin is poised for further growth in 2013 and beyond.
2012 was a milestone year for BioMarin on a number of important fronts. We announced positive results for our Phase III Vimizim™ study for Morquio A syndrome, and expect to file for approval in the US, EU and other key markets in 2013. We also announced positive results from our PEG-PAL Phase II study in PKU and plan on initiating a Phase III study in 2013. We reached the half billion dollar revenue level and expect to see revenues from our first-in-class commercial therapies continue to grow.
Time is critical to patients with rare diseases who have few, if any, treatment options, and we strive to quickly develop important therapies for them. The efficiency and speed of our research, development, manufacturing, and commercial efforts is at the heart of our ability to urgently deliver therapies. Our track record of developing and commercializing new treatments, which is on average four and one half years per therapy, has been significantly faster than the industry average of eight to ten years. This is a remarkable accomplishment that is engrained in our culture.
We continue to look for innovative technologies and molecules to grow our pipeline for our future and fulfill our commitment to patients suffering from rare diseases. We are world leaders in metabolic disease innovation with three approved treatments: Naglazyme®, Alduarzyme® and Kuvan®; and an anticipated approval of Vimizim. Our experience developing treatments for MPS diseases, and potentially a novel treatment for achondroplasia demonstrate our world class expertise in treating skeletal dysplasias.
We continue to fuel our R&D engine with opportunities that play to our strengths and competencies. We relentlessly pursue exciting, early-stage science that has the potential to change the course of disease. I am particularly excited about the future of our pipeline as BioMarin builds on its research accomplishments by pioneering two new technology platforms: glycobiology and gene therapy.
Gene therapy has the potential to be a disruptive technology that not only holds the promise of better treatments for patients, but to revolutionize the practice of medicine. We licensed a Factor VIII gene therapy research program for hemophilia A from University College London and St. Jude Children’s Research Hospital. Hemophilia A is an attractive target for gene therapy, and BioMarin is now at the forefront of this novel technology, which is emerging as a viable way to treat genetic disorders.
In addition, our acquisition of Zacharon Pharmaceuticals will further expand our glycobiology expertise and support our drug development efforts in lysosomal storage diseases. The acquisition strengthened our preclinical pipeline with two programs in lead optimization: heparan sulfate inhibitors for MPS III and ganglioside synthesis inhibitors for gangliosidoses, such as Tay Sachs and Sandhoff. Glycobiology also will provide us with an important clinical diagnostic to measure a patient’s response to therapy and to adjust that therapy for the best individual outcome.
Making a Difference
As we embark on 2013, we are proud of the foundation that we have established and the difference we are making to patients suffering from rare genetic diseases. Today, our pipeline continues to be sustained by our ongoing commitment to R&D investment. This long-term investment is paying off for patients, employees and investors. Beyond product approvals and clinical milestones, we have also established our manufacturing capabilities and built a global commercial infrastructure that stands ready to launch Vimizim. We are inspired and driven by the patients who receive our therapies, and we will continue our efforts to help more patients suffering from rare diseases who have unmet medical needs.
BioMarin is in a transformative phase of its history. Our success stems from our unwavering focus on science and the hope our therapies can bring to patients. I wish to thank all of the patients and physicians who help us develop breakthrough therapies. I also would like to thank our employees who make it possible for BioMarin to deliver therapies that are critical to patients with rare genetic diseases.
Thank you for your support.
Chief Executive Officer
BioMarin Pharmaceutical Inc.