A MESSAGE FROM THE CEO
Welcome! Thank you for visiting the BioMarin Pharmaceutical website.
BioMarin leads the biotechnology industry in delivering therapies for chronic and degenerative genetic conditions that reduce patients' quality of life and shorten life spans. We target diseases that lack effective therapies and affect relatively small numbers of patients, many of whom are children. As we enter 2014, BioMarin will continue to focus on advancing therapies that are the first or best of their kind and make a big difference in improving patients' lives.
The last 18 months have been a phenomenal time for BioMarin. With the approval of VIMIZIM®(elosulfase alfa), the first and only specific treatment for Morquio A or MPS IVA, we fulfilled a significant unmet medical need for patients and their families and firmly established our leadership in developing therapies to treat MPS diseases.
With VIMIZIM, we believe we can bring meaningful and unprecedented benefits to patients related to improvement in basic activities of daily living, such as walking or time away from a wheelchair. Importantly, as we pursue the commercial launch of VIMIZIM in the United States and beyond, we will leverage our established, targeted commercial footprint built over the last seven years to support Nagalyzme®(galsulfase).
Our MPS franchise is now comprised of three commercial products – VIMIZIM, Aldurazyme® (laronidase), and Naglazyme – all of which are enzyme replacement therapies to treat lysosomal storage disorders. Building on this expertise, earlier this year we added BMN 250 for Sanfilippo B or MPS IIIB to our pipeline and expect to move the molecule into the clinic next year. Also new to the pipeline in 2014 is BMN 270, a gene therapy treatment for hemophillia A. We are excited about gene therapy because of its potential to be a one-time treatment that could completely disrupt the current standard of care for patients.
We reached many important clinical milestones in 2013 and 2014. We enrolled the first patient in our Phase 3 PEG PAL trial, which represents a potentially exciting option for PKU patients who do not achieve sufficient benefit from our current therapy, KUVAN® (sapropterin dihydrochloride). We initiated a Phase 3 trial with BMN 673 for germline BRCA breast cancer. The data from the Phase 1/2 trial looks promising, and we believe BMN 673 could possibly help a small subset of breast cancer patients by providing a targeted treatment option.
Looking forward, 2014 will be the year we move multiple clinical programs forward and expand our early stage pipeline. We will begin enrollment in our Phase 2/3 trial for BMN 701 for Pompe disease and continue enrollment in both our Phase 2 trials for BMN 111 for achondroplasia, a form of dwarfism, and our Phase 1/2 trial for BMN 190 to treat children with of Batten disease. The particular form of Batten disease that we are targeting affects a few hundred children in the developed world and is so devastating that the children who suffer from it rarely live past the age of 12.
We now have five approved commercial products, and the addition of VIMIZIM to our MPS franchise has the potential to double our revenues to one billion dollars. We continue to support and invest in our five approved commercial products. For example, at the end of 2013, the FDA approved a new and convenient powder form of KUVAN that provides an alternative to tablets and offers an additional treatment option for infants and small children.
To meet our future commercial and clinical product needs, manufacturing continued to scale its operations to meet long-term anticipated demand. Infrastructure improvements within our Novato, California manufacturing facility continue to add both cell culture and fermentation capacity to support our seven clinical and commercial biological products. Our Novato manufacturing facility is capable of supporting demand until our plant in Shanbally, Ireland is fully operational, which is expected in 2017. Further, the FDA approval of VIMIZIM in February 2014 included the approval of our quality control laboratories at our Shanbally facility .
The treatments that we are developing or marketing help patients who are mostly children and have few, if any, options to treat their diseases. We are grateful to the patients and physicians who participate in clinical trials to test experimental therapies for rare genetic diseases. Every part of our organization is poised to drive our business forward from the discoveries in the lab to the launch of new products.
We thank you for your continued support and look forward to generating further value throughout 2014.
Thank you for your support.
Chief Executive Officer
BioMarin Pharmaceutical Inc.