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For years, BioMarin CEO Jean-Jacques “J.J.” Bienaime has been saying that Roctavian — the company’s gene therapy to treat hemophilia A — could be the biggest product the biotech firm has ever launched. Based on a meeting with STAT Sunday, his hopes don’t seem to have dimmed a bit despite the regulatory delays the product has faced in the U.S.

“I think over time, this product is going to be sold by the patients that are taking it,” Bienaime told STAT while sipping an espresso in a posh San Francisco hotel. “When you talk to people who were in our pivotal trials, whether Phase 2 or Phase 3, that have been treated for several years, this is life-changing for them. And I think they will communicate that to the patient community.”


BioMarin said in a press release Sunday that it now has data following individuals in a 134-patient study for four years after treatment, and that their annualized bleeding rate has decreased 80% during that time. BioMarin said that 92% of patients no longer were taking clotting factors regularly to prevent bleeding in their bodies, particularly in their joints.

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