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June, 2023 Quick Question Review

Our June 2023 Quick Question asked, “Name the bispecific antibody-based FVIII inhibitor bypass agent.” Our 68 respondents answered…

  1. Caplacizumab [Cablivi]: 3 [4%]
  2. Eculizumab [Soliris]: 8 [12%]
  3. Emicizumab [Hemlibra]: 53 [78%]
  4. Eloctate: 3 [4%]
  5. Jivi: 1 [2%]

Genentech’s emicizumab is the bispecific antibody that bypassed FVIII in to join FIX to FX, activating FX. Emicizumab is available to all hemophilia A sufferers including those with anti-VIII inhibitors.

Caplacizumab is Sanofi’s VWF-specific nanobody designed to treat TTP by suppressing the activity of ULWF. Caplacizumab is the first nanobody-based therapeutic.

Eculizumab is Alexion’s complement factor V antibody first designed to treat paroxysmal nocturnal hemoglobinuria [PNH] and is now approved to treat atypical hemolytic-uremic syndrome [aHUS}.

Eloctate [Sanofi] and Jivi [Bayer] are two examples of the several extended half-life FVIII concentrates available since 2014 that are routinely used for hemophilia A prophylaxis.

Not listed in the question is Novo Nordisk’s  concizumab, an anti-TFPI antibody therapeutic currently under FDA review that may become available to treat hemophilia A and B.

 

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