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“NEW YORK (Reuters) – BioMarin Pharmaceutical Inc. said U.S. regulators approved its drug to treat a rare, often fatal genetic disease and granted it orphan drug status, boosting its shares by more than 9 percent on Wednesday.
The drug, Naglazyme, is an enzyme replacement therapy for a disease called mucopolysaccharidosis VI. BioMarin said it expects to begin selling the drug in about 30 days.
The U.S. authorities granted BioMarin seven years of marketing exclusivity in the United States.
The biotechnology f rm’s Swiss-listed shares rose 9.6 percent to 8.89 francs by 0758 GMT, adding to a 13 percent gain so far this year.
“The approval is good for the company and we expect a positive influence on today’s share price,” Bank Leu said in a note.
BioMarin said it was awaiting a decision on European approval for Naglazyme.
MPS VI is caused by an enzyme deficiency and leads to progressive cellular, tissue and organ system dysfunction.
In clinical trials, the drug demonstrated an ability to improve endurance in patients who undertook 12-minute walk and 3-minute stair climb tests.
The U.S. Food and Drug Administration grants its orphan drug designator to treatments that may offer significant benefit in patients with serious or life threatening diseases that occur in not more than 200,000 patients.
Only an estimated 1,100 individuals in the developed world suffer from MPS VI, BioMarin said. Most people with the condition die from disease-related complications between childhood and early adulthood. (Additional reporting by Josiane Kremer in Zurich)