BioMarin Pharmaceutical Inc. (BMRN) Friday said it has received approval from the U.S. Food and Drug Administration, or FDA, for its VIMIZIM, indicated for treatment in patients with Mucopolysaccharidosis type IVA.
"We have developed three therapies to treat three different MPS diseases and continue to build on our extensive scientific and clinical knowledge of lysosomal storage disorders to develop therapies for other rare genetic diseases, " said Jean-Jacques Bienaimé, CEO.
"In clinical trials, VIMIZIM was shown to significantly improve endurance, which possibly could change the course of the disease. As a treating physician, I am encouraged that the therapy has proven to provide clinical benefit, which is not always possible to demonstrate with ultra-rare diseases," said Paul Harmatz, M.D., clinical investigator in the VIMIZIM Phase 3 trial.
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