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Positive Opinion From EMA for BioMarin’s VIMIZIM for Morquio A Syndrome

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Biomarin logo total orphan drugs

February 20, 2014

BioMarin Pharmaceutical Inc. announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the company’s Marketing Authorization Application (MAA) for VIMIZIM™ (elosulfase alfa) for the treatment of Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA). The CHMP’s recommendation is now referred to the European Commission (EC). If approved by the EC, BioMarin would receive marketing authorization for VIMIZIM in all EU Member States. The EC is expected to render a final decision for VIMIZIM in the second quarter of 2014.

“This positive CHMP opinion is an important milestone in our mission to provide the first approved therapy to treat Morquio A patients across Europe. We will leverage our existing European infrastructure to ensure that these patients gain access to VIMIZIM as quickly as possible,” said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. “We are grateful for the continuous support we have received from the Morquio A community, and in particular, those patients who participated in the clinical development of VIMIZIM and their families.”

“As a treating physician, the CHMP opinion is meaningful news for Morquio A patients who currently have no specific drug treatment option beyond supportive care,” said Christian J. Hendriksz, Salford Royal NHS Foundation Trust. “VIMIZIM improved endurance in clinical trials, which may change the course of this devastating disease.”

The U.S. Food and Drug Administration (FDA) approved VIMIZIM for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome) on February 14, 2014.

VIMIZIM is an enzyme replacement therapy for the treatment of patients with the lysosomal storage disorder Morquio A syndrome, which is an ultra-rare, severely debilitating disease that affects an estimated 3,000 patients in the developed world. VIMIZIM is the first approved drug treatment for Morquio A syndrome.

The disease occurs as a result of a deficiency of activity in an enzyme involved in glycosaminoglycan (GAG) metabolism. The pervasive and progressive accumulation of GAGs leads to significant morbidities and multisystemic clinical impairments resulting in diminished functional capacity, impaired quality of life, and early mortality. The most common features of the disease are progressive skeletal dysplasia, the need for frequent surgical procedures related primarily to musculoskeletal or respiratory dysfunction, and significant limitations in mobility, endurance, and breathing.

The CHMP is a scientific committee composed of representatives from the 28-member states of the EU, and Iceland and Norway. The committee reviews medical product applications on their scientific and clinical merit and provides advice to the EC, which has the authority to approve medicines for the EU. The EU, which generally follows the recommendation of the CHMP, is expected to make its final decision in about 60 days.

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