A MESSAGE FROM THE CEO
Welcome! Thank you for visiting the BioMarin Pharmaceutical website.
BioMarin is a world leader in providing therapies to treat patients with rare diseases. We have five products available to patients and one of the most diverse and exciting product pipelines in our industry. True to our origins as pioneers almost two decades ago, we continue to blaze scientific trails on behalf of children and adults with rare genetic diseases.
In 2014, our continued revenue growth reflected the value that our therapies offer to our patients. Total revenues grew 37 percent from the previous year to $751 million. The successful launch of Vimizim, an enzyme replacement therapy (ERT) for Morquio A Syndrome or MPS IVA, delivered $77.3 million in net product revenues in just under a year. Our experience in developing, marketing and manufacturing ERTs laid the groundwork for the most successful product launch in BioMarin’s history. We also advanced all of our clinical development programs during the year. In early 2015, we acquired Prosensa and added multiple potential products to treat Duchenne Muscular Dystrophy (DMD), including drisapersen, an investigational therapy for the largest genetically defined subgroup of this patient population.
At the end of April, we submitted a New Drug Application to the United States Food and Drug Administration for drisapersen. Drisapersen induces the skipping of dystrophin exon 51, potentially providing a therapeutic benefit to approximately 13% of DMD patients. DMD is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births with about 20,000 new cases diagnosed each year. We believe that drisapersen has the potential to be the first-to-market treatment option, which could make a meaningful difference for boys with Duchenne. We also intend to submit an application for registration in the European Union this summer.
In early 2015, we announced promising interim data from a Phase 1 trial of cerliponase alfa for the treatment of CLN2 disease, a form of Batten disease. The initial data suggests that the treatment may be able to slow the disease’s progression compared to its devastating natural history, which robs the lives of children around the age of 12. This trial reflects BioMarin’s fundamental commitment to children and their families with fatal rare diseases and no treatment options, and the trial’s encouraging progress demonstrates our scientific acumen to deliver an enzyme replacement therapy directly to the brain.
As we look ahead to 2015, BioMarin has ten products in various stages of development and is well-positioned for a transformational year of value creation. Our clinical and commercial programs are supported by BioMarin’s world-class manufacturing capabilities. We continue to build our manufacturing capacity both to meet the demand for our commercial products and the products in clinical trials.
We plan to enter Phase 1 clinical trials in BMN 270 in gene therapy for hemophilia A and in BMN 250 for Sanfilippo B syndrome or MPS IIIB. Gene therapy represents a potentially disruptive technology that could change the practice of medicine because it could allow for one treatment only, compared to lifelong treatments. BMN 250 has the potential to be the fourth therapy in our MPS franchise and is supported by knowledge gained from other products in our portfolio, including our experience in delivering enzymes directly to the brain.
May 2015 will mark the tenth anniversary of Naglazyme’s U.S. approval, which is the wellspring of our subsequent achievements. The original MPS VI natural history study conducted in 2003 showed patients with the rapidly progressing form of MPS VI were not likely to live into their twenties. We are now working with physicians and patients to face the challenges of treating adult patients who desire normal experiences of being an adult, such as college and a career. We are grateful to be confronting such a challenge.
Due to our success and rapid growth, we are a very different company from the one that launched Naglazyme a decade ago, yet we have remained true to our core – to make a big difference for small patient populations. We still know many of our patients by name and regularly spend time with them at patient gatherings or at events on our campus. We continue to be inspired by strong science, and when we see opportunities where the biology of the disease is well understood, we take calculated risks.
While the science builds the platform for clinical success, we could not do what we do without a commitment to long-term relationships with patient communities, physicians, academic researchers, our employees, elected officials, regulatory bodies, investors and the local communities where we operate. The patients and families that we serve are extraordinary, and we appreciate their participation in the clinical trials required to provide approved treatments. The optimism, resiliency and thoughtfulness of our patients motivate us to deliver breakthrough therapies that will change the course of genetic diseases.
Chief Executive Officer
BioMarin Pharmaceutical Inc.