BioMarin

BioMarin Pharmaceutical develops enzyme therapies for serious, life-threatening diseases and conditions. We are committed to improving the lives of patients by developing novel enzyme therapies for currently unmet medical needs. BioMarin is currently sponsoring a clinical trial of enzyme replacement therapy for MPS IVA (Morquio A). A natural history study of MPS IVA, sponsored by BioMarin, is underway in the United States and several other countries.

BioMarin Announces FDA Approval for Naglazyme

In May 2005, Naglazyme received marketing approval from the U.S. Food and Drug Administration (FDA) for the treatment of individuals with MPS VI. Shortly thereafter, in January 2006, Naglazyme received approval in the European Union. Naglazyme was designated an orphan drug in the US and EU, which grants market exclusivity for 7 and 10 years, respectively. Naglazyme has also been approved in Australia, Brazil, Croatia, Japan, South Korea, Switzerland, and Belarus. Naglazyme has been granted orphan drug designation in Australia, Japan, South Korea and Switzerland as well. Naglazyme has been shown to improve walking and stair-climbing capacity. Naglazyme is available by prescription only. To learn more, please visit www.naglazyme.com for full prescribing information.

BioMarin Announces FDA Approval for Vimizim

In February 2014, BioMarin  announced that the U.S. Food and Drug Administration (FDA) has approved VIMIZIM™ (elosulfase alfa) for patients with MPS IVA, Morquio A syndrome. VIMIZIM is an enzyme replacement treatment for Morquio A syndrome, which affects an estimated 3,000 patients in the developed world.   Absence of this enzyme leads to problems with bone development, growth and mobility. The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism. Vimizim is intended to replace the missing GALNS enzyme involved in an important metabolic pathway and improve endurance in patients.  Vimizim was granted priority review. An FDA priority review provides for an expedited review of drugs for serious diseases or conditions that may offer major advances in treatment. Vimizim is also the first drug to receive the Rare Pediatric Disease Priority Review Voucher – a provision that aims to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. For more information about VIMIZIM, please visit www.VIMIZIM.com.

BioMarin Offers Help with Insurance Questions for MPS VI

BioMarin has developed a free and confidential service designed to help assist with healthcare insurance questions. The BioMarin Patient and Physician Support program (BPPS) can help families evaluate their current healthcare insurance coverage, provide information on potential healthcare insurance options that may be available in your state, and educate insurance companies about MPS VI. To contact a BPPS patient advocate, please call their toll free number: (866) 906-6100.