Big U.S. pharma stocks are up about 5 percent so far this year. Biotechs, meanwhile, are up nearly 6 percent and have risen 17 percent over the past six months.
Jean-Jacques Bienaime, BioMarin Pharmaceutical's CEO, told CNBC his company has five products in clinical trials, which is large for a company its size.
BioMarin is most excited about a treatment for Morquio syndrome, or MPS IV, an enzyme deficiency that can lead to skeletal dysplasia, short stature, and joint abnormalities.
"It has the potential of doubling the revenues of the company and bringing it to about a billion dollars of revenues in the future," Bienaime said of the treatment. He expects to file for approval with the Food and Drug Administration by the end of the quarter and with European authorities by the end of April.
"There's a good chance to get approval this year," he added.
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