BioMarin Announces FDA Approval for VIMIZIM(TM) (elosulfase alfa) for the Treatment of Patients With Morquio A Syndrome

BioMarin Announces FDA Approval for VIMIZIM(TM) (elosulfase alfa) for the Treatment of Patients With Morquio A Syndrome

FDA approves Vimizim to treat rare congenital enzyme disorder 

First drug to receive Rare Pediatric Disease Priority Review Voucher

The U.S. Food and Drug Administration today approved Vimizim (elosulfase alfa), the first FDA-approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome). Morquio A syndrome is a rare, autosomal recessive lysosomal storage disease caused by a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Vimizim is intended to replace the missing GALNS enzyme involved in an important metabolic pathway. Absence of this enzyme leads to problems with bone development, growth and mobility. There are approximately 800 patients with Morquio A syndrome in the United States.

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.

The safety and effectiveness of Vimizim were established in a clinical trial involving 176 participants with Morquio A syndrome, ranging in age from 5 to 57 years. Participants treated with Vimizim showed greater improvement in a 6-minute walk test than participants treated with placebo. On average, patients treated with Vimizim in the trial walked 22.5 meters farther in 6 minutes compared to the patients who received placebo.
 
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm386008.htm
 
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