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Reports US

US stock market daily report (February 18, 2014, Tuesday)

February 19, 2014, Wednesday, 04:46 GMT | 00:46 EST | 10:16 IST | 12:46 SGT
Contributed by Millennium Traders

BioMarin Pharmaceutical Inc. (BMRN-Nasdaq) announced approval from the U.S. Food and Drug Administration on February 14 for Vimizim (elosulfase alfa), the first FDA approved treatment for Mucopolysaccharidosis Type IVA (Morquio A syndrome). BioMarin received priority review for Vimizim from FDA. 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. European drug regulators are expected to render an approval opinion for Vimizim on Friday. Morquio syndrome is a rare genetic disorder that mainly affects the skeleton due to a deficiency in N-acetylgalactosamine-6-sulfate sulfatase (GALNS) which leads to problems with bone development, growth and mobility. The missing GALNS enzyme involved in an important metabolic pathway are intended to be replaced by Vimizim.

On Tuesday, BioMarin reported the annual cost for treatment with Vimizim at nearly $380,000. In the United States there are approximately 800 patients with Morquio A syndrome and around the world there are approximately 700 patients.

Andrew E. Mulberg, M.D., deputy director, Division of Gastroenterology and Inborn Errors Products in the FDA’s Center for Drug Evaluation and Research (CDER) said, “This approval and rare pediatric disease priority review voucher underscores the agency’s commitment to making treatments available to patients with rare diseases. Prior to today’s approval, patients with this rare disease have had no approved drug treatment options.”

Clinical trial for Vimizim involved 176 patients with Morquio A syndrome, ranging in age from 5 to 57 years. Patients participating in the trial, who received Vimizim, showed greater improvement in a 6-minute walk test than participants treated with placebo. Patients treated with Vimizim in the trial, on average, walked 22.5 meters farther in 6 minutes compared to the patients who received placebo.

The most common side effects in patients treated with Vimizim during clinical trials included fever, vomiting, headache, nausea, abdominal pain, chills and fatigue. The safety and effectiveness of Vimizim have not been established in pediatric patients less than 5 years of age. Vimizim is being approved with a boxed warning to include the risk of anaphylaxis. During clinical trials, life-threatening anaphylactic reactions occurred in some patients during Vimizim infusions.