PRIME designation for Hemophilia B gene therapy

uniQure today announced that it received Priority Medicines (PRIME) Designation from the European Medicines Agency for its Hemophilia B gene therapy called ATM-060.

PRIME designation is the EMA’s equivalence of Breakthrough Therapy Designation granted by the FDA.

“This designation is based on results from the ongoing, dose-ranging Phase 1-2 study that show a near cessation of spontaneous bleeding in patients with severe disease at up to 12 months follow-up, clinically significant and sustained increases in Factor IX (FIX) and substantial reductions in FIX replacement usage.”

UniQure had reported clinical data from the ongoing Phase 1-2 trial of AMT-060 last December at ASH. Data from the second-dose cohort had shown a dose response with substantial improvement in disease state in five treated patients.

The company has developed a proprietary non-replicating AAV-based Vector Delivery System to deliver the desired gene cassette.

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