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
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