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UniQure Hemophilia B Gene Therapy Changes Ignite A Race To Market

 
• By Jessica Merrill

UniQure is switching the gene therapy it plans to move into a pivotal trial in hemophilia B patients because the new version could have clinical benefits. Regulatory agencies in the US and Europe have given the change a green light, and uniQure plans to begin a pivotal trial in 2018.

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UniQure NV is putting rivals Spark Therapeutics Inc./Pfizer Inc. to the test when it comes to the race to bring the first gene therapy for hemophilia B to market. The company has gotten a green light from the US FDA and European Medicines Agency to move forward with a pivotal trial testing a new version of the therapy.

In back-to-back announcements Oct. 19, the Netherlands-based gene therapy developer said it has made a big change to its lead program but that FDA and EMA are comfortable with the change

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