STAT+: BioMarin executives express confidence around hemophilia gene therapy

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.”

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