Message from Management
Welcome! Thank you for visiting the BioMarin Pharmaceutical website.
2010 was a key, foundation-building year in the next phase of growth for BioMarin. Through the efforts of our passionate and hard working employees, we have expanded our commercial opportunities while continuing to advance key pipeline programs that have the potential to shape the company in the near future.
Increasing Opportunities
Highlights of the year include strong sales of our commercial products, the launch of Firdapse™ (amifampridine phosphate) for LEMS in Europe, the acquisitions of LEAD Therapeutics and ZyStor Therapeutics which added promising compounds to our pipeline and the continued advancement of several clinical and non-clinical programs. The company’s total net revenue increased 16% over 2009, posting at $376.3 million. Naglazyme sales in new and existing markets continued strong growth with a 14% increase in net product sales over 2009, and third party sales of Aldurazyme showed modest gains as the number of patients continued to increase. Total annual net revenues of Kuvan were $99.4 million, a 29% increase compared to 2009. Firdapse revenues from launch in April to the end of 2010 were $6.4 million. Year-end cash, cash equivalents and short and long term investments posted at $402.3 million for the year. All the while, we continue to expand our global commercial reach, treating patients in over 40 countries.
Expanding Portfolio
Our diversified R&D efforts continue to solidify our position in the rare disease arena. Important progress was made in our most advanced clinical programs, as we recently began the pivotal Phase III trial for GALNS for MPS IVA, which will be the largest enzyme replacement trial ever conducted. Encouraging data from the PEG-PAL phase II trial for PKU, completed in mid-2010, has led to a Phase IIb trial to determine optimal dosing, with an anticipated pivotal phase III trial to commence in early 2012. Our pipeline continues its strong growth as demonstrated by our filing INDs for both BMN-673 (PARP inhibitor) and BMN-701 (Pompe disease), with both programs beginning phase I trials in early 2011. We also announced our CNP program for Achondroplasia, for which we expect to file an IND in late 2011.
We continue to build important data on Kuvan therapy outcomes, initiating a phase IIIb study on neuropsychiatric symptoms in PKU patients and expect the commercial availability of a handheld blood Phe monitoring device late this year. Additionally, we have moved forward with development plans for Firdapse in the U.S., filing an IND for this program with the FDA in late 2010.
Continuing Progress
The pipeline achievements and commercial performance of 2010 have positioned BioMarin to meet our goals for 2011 and beyond. As we look forward to the year ahead, we are focused on delivering on the promise of our growing pipeline, while at the same time maintaining double digit revenue growth that will continue to drive maximum long term value to patients and our shareholders.
I thank all of our employees for their expertise, passion and dedication to our mission throughout the year. I would also like to thank the patients and physicians who partner with us to treat rare diseases. I look forward to keeping you informed and sharing future developments during, what we believe will be a pivotal year in the growth of BioMarin.
Thank you for your support.
Sincerely,
Jean-Jacques Bienaimé
Chief Executive Officer
BioMarin Pharmaceutical Inc.
