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Ahead of schedule FDA approves Vimizim to treat rare disease

By Peter Winter, Editor

While most investors had their eyes glued all-day to the FDA’s website awaiting a scheduled decision on Chelsea Therapeutics International Ltd.’s droxidopa (Northera) it surprised everyone by giving the green light to Novato, Calif.-based Biomarin Pharmaceutical Inc.’s Vimizim (elosulfase alfa), two weeks ahead of its PDUFA date of Feb 28.

Vimizim, according to the agency represents 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 iand whose absence leads to problems with bone development, growth and mobility. There are approximately 800 patients with Morquio A syndrome in the U.S.

While the early approval caught many by surprise, it wasn’t entirely unexpected. 

The surprising FDA decision came so late in the day that the company’s share price wasn’t affected closing the day at $75.78, down 15 cents.