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SAN RAFAEL, Calif., Nov. 19, 2013 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced that the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) of the U.S. Food and Drug Administration (FDA) voted in favor of approval of Vimizim for the treatment of Morquio A syndrome, also called Mucopolysaccharidosis Type IVA (MPS IVA). Of the 21 panel members, 19 voted in favor of approval of Vimizim for use in all MPS IVA patients, 1 voted in favor of approval for a subgroup of MPS IVA patients, and one panel member voted to not recommend approval.
Vimizim is an investigational enzyme replacement therapy for the treatment of patients with the lysosomal storage disorder Morquio A syndrome. Morquio A syndrome is an ultra-rare, severely debilitating disease that affects an estimated 3,000 patients in the developed world.
"We are thrilled to have achieved this important milestone in our mission to bring the first approved therapy to treat Morquio A patients," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "I am very pleased with the outcome of today's panel vote and look forward to continuing to work with the FDA to bring this much-needed therapy to these patients."
The FDA has assigned a Prescription Drug User Fee Act (PDUFA) action date of February 28, 2014, for completion of its review of the Biologics License Application (BLA) for Vimizim. The EMDAC provides the FDA with independent expert advice and recommendations. The FDA is not bound by the EMDAC's recommendation, but will consider the committee's recommendation as the FDA completes its review of the Vimizim BLA.
VIMIZIM (elosufase alfa) is an investigational treatment for patients with Morquio A syndrome, or mucopolysaccharidosis IVA (MPS IVA), which currently is under review by the FDA. VIMIZIM is the first and only enzyme replacement therapy (ERT) designed to target the underlying cause of Morquio A Syndrome – a deficiency in the enzyme N-acetylgalactosamine-6 sulfatase (GALNS). Infused enzyme replacement therapy with VIMIZIM replaces deficient GALNS activity to minimize progressive multi-systemic manifestations. Morquio A syndrome is a rare, severely debilitating and progressive disease that currently has no standard accepted treatment other than supportive care.