Hemophilia Therapies: Factor Replacement and Emicizumab


Lisa Schrade, PharmD, discusses factor replacement therapies and emicizumab’s role in treating hemophilia.

Jonathan Ogurchak, PharmD, CSP: The therapies for hemophilia [are] critical to help prevent bleeds [and] stop all of these life-threatening episodes. Dr Schrade, could you talk a little bit about some of the replacement treatments that are available as far as factor replacement, as well as drugs like emicizumab?

Lisa Schrade, PharmD: Currently [in the] standard of care, you have your standard half-life products, which have a half-life between 8 and 12 hours. Their glycoproteins can be plasma derived or produced from recombinant therapy technology, and what they’re supposed to do is help replace the missing factor 8 to aid with restoring hemostasis. The next therapy line would be the extended half-life products. Those have molecules that have been modified in some way, some like PEGylation or fusion protein to help delay the degradation of the factor protein in the body so it lasts longer and has higher levels. In theory, the extended half-life products should need less frequent infusions compared [with] the standard half-life products. You also have bypassing agents, which we normally see reserved for patients with inhibitors as we’re trying to tolerize and overcome that inhibitor.

They’re not as effective as factor products, and there’s definitely the burden of administration because you’re infusing more frequently, like every 2 hours, as opposed to the standard half-life that might be 3 to 4 times a week, and then the extended half-life products, which are 1 and a half times longer for their half-life than the standard half-life products. Emicizumab is known as a mimetic factor 8 agent because it mimics or imitates the activity of factor 8 in the system by bridging factor 9 and factor 10 so that we can help restore the function of the clotting cascade.

Transcript was AI generated and reviewed by a Pharmacy Times® editor.

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