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BioMarin’s Gene Therapy Nears European Market After US Setback

  • US drugmaker seeks to expand where Bluebird Bio stumbled
  • Reimbursement pacts could take a year to reach, BioMarin says
Jeff Ajer
Jeff AjerSource: BioMarin

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 Commerical 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.