Biomarin’s hemophilia gene therapy nears European market after US setback

A single dose of the US-based biotech firm’s drug could be enough for a lifelong treatment of the rare bleeding disorder – but Roctavian’s price tag is making governments around the world think twice.

Photo: Peter Dejong/AP/Ritzau Scanpix

Biomarin Pharmaceutical Inc. expects European patients to begin receiving its gene therapy for a life-threatening blood-clotting disorder before the end of the year, raising the drugmaker’s hopes it can succeed where rival Bluebird Bio Inc. faltered.

The one-time infusion for hemophilia could gain European approval at the end of August after getting a key recommendation in June from a regulatory committee, Biomarin Chief Commercial Officer Jeff Ajer said in an interview. The company will then make a country-by-country push to persuade health systems to pay for it, starting with Germany, with broader agreements likely taking about a year to clinch, he said.

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