Based on the positive data, Biomarin now intends to advance the gene therapy, known as BMN 270, into a new study designed to support an accelerated approval in the U.S. and Europe.
BMN 270 is one of the most important drugs in Biomarin's research pipeline and key driver of future revenue and earnings.
Near the end of Wednesday's regular trading session, Biomarin shares rose 6% to $97.56.
If approved, BMN 270 would be the first gene therapy used to treat -- and potentially cure -- patients with hemophilia A, a genetic disorder caused by missing or defective factor VIII, a clotting protein.
In the study presented Wednesday, nine patients with severe hemophilia A received a single dose of BMN 270, seven at the highest dose. Six of the seven patients had Factor VIII levels above 50%, which is nearly normal. The annualized bleeding rate for all seven of the high-dose patients also fell.
There were no serious safety events reported in the study.
"These data provide strong proof of concept evidence that restoration of clotting function may be achieved by gene therapy," said John Pasi, professor of Haemostasis and Thrombosis at Barts and the London School of Medicine and Dentistry and primary investigator for the BMN 270 study, in a statement. "For the first time, patients have reason to hope to avoid bleeding and the opportunity to live a normal life."
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