First Treatment for MPS IVA Approved

February 14, 2014

Dear Friends:

BioMarin Pharmaceutical announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Vimizim™ (elosulfase alfa), the first specific therapy approved for the treatment of Morquio A (MPS IVA syndrome.)

“The FDA approval of VIMIZIM is an important milestone for BioMarin and for patients with Morquio A syndrome. VIMIZIM is the first and only therapy designed to address the condition at the cellular level, fulfilling a large unmet medical need for patients and their families,” said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. “With the approval of VIMIZIM, BioMarin firmly establishes its leadership in advancing therapies to treat MPS diseases. We have developed three therapies to treat three different MPS diseases and continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases.”

The National MPS Society is proud to have played a part in this exciting and historic event.  As we reported in November, the Society worked closely with BioMarin to prepare for the FDA Advisory Committee meeting.  A huge thanks to the individuals and their families who spoke before the committee, ensuring their YES vote – Maurizo Bellassai, Stephanie Bozarth, Mary Cavanagh, Nick Gaudin, Doug Kreul, Sarah Van Orden, and Steve Holland.

The National MPS Society thanks BioMarin for their commitment to the MPS community.  Vimizim™ is the fourth FDA approved treatment for an MPS disease, and the third that BioMarin has developed.  However, the Society will NOT rest until there are treatments and cures for ALL MPS and related diseases.

Our fight for you continues.

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