FDA Approves UniQure's Hemgenix, First Hemophilia B Gene Therapy For Adults

UniQure N.V.'s QURE partner CSL Limited CSLLY has received FDA approval for Hemgenix (etranacogene dezaparvovec-drlb), a one-time gene therapy for hemophilia B.
CSL licensed the exclusive global rights to Hemgenix from uniQure in May 2021.
UniQure has received about $500 million so far from CSL and will also be eligible to get up to an additional $1.5 billion in commercial milestone payments and royalties.
Cowen analyst Joseph Thome said that Hemgenix's price of $3.5 million is higher than the brokerage's estimated $1.9 million, but its consultants have been optimistic about the therapy's uptake, Reuters reported.
Results from the pivotal HOPE-B trial support the FDA approval, the largest gene therapy trial in hemophilia B.
Results from the study demonstrated that Hemgenix allowed patients to produce mean factor IX activity of 39% at six months and 36.7% at 24 months post-infusion.
Seven to 18 months post-infusion, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by 54% compared to the six-month lead-in period on factor IX prophylactic replacement therapy (4.1 to 1.9).
In addition, 94% of patients treated with Hemgenix discontinued prophylaxis and remained free of previous continuous routine prophylaxis therapy.
The FDA has already cleared two gene therapies, Zynteglo and Skysona, from Bluebird bio Inc BLUE in August and September, respectively.
While Zynteglo was priced at $2.8 million, Skysona had a wholesale cost of $3.0 million.
Price Action: QURE shares closed 1.74% higher at $23.40 during after-hours trading on Tuesday.
Photo Via Company