UniQure’s haemophilia B therapy picks up PRIME status

uniQure’s investigational gene therapy for patients with the blood clotting disorder haemophilia B has been awarded PRIME status by European regulators, potentially placing the treatment on a faster path to approval.

The European Medicines Agency introduced its PRIME programme last year to expedite the regulatory process for investigational medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options.

uniQure’s AMT-060 was accepted onto the scheme on the back of data from an ongoing, dose-ranging Phase I/II study that has 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, the firm said.

Also, on the safety side, the therapy continues to be well-tolerated in the trial, and there have been no severe adverse events. Three out of a total of 10 patients have thus far experienced mild, asymptomatic elevations of the liver enzyme alanine aminotransferase (ALT), but these were not associated with any loss of endogenous FIX activity or T-cell response, it was stressed.

"We are very pleased to have AMT-060 for haemophilia B accepted into the PRIME program," stated Matthew Kapusta, chief executive officer of uniQure. "Similar to the Breakthrough Therapy designation that AMT-060 received from the U.S. Food and Drug Administration earlier this year, we look forward to this enhanced collaboration with the EMA to advance the clinical development of  this potentially transformative therapy for haemophilia B patients."

Haemophilia B is a rare, inherited disorder occurring in around 1 out of 30,000 live births. Current treatment consists of prophylactic or on-demand protein replacement therapy, in which one to three times weekly intravenous administrations of plasma-derived or recombinant hFIX are required to prevent bleeding and once daily infusions in case bleeding occurs.