As the understanding of BPDCN biology has grown, newer therapies have emerged that focus on specific molecular targets to improve effectiveness and reduce side effects. 

Targeted therapy uses medicines to focus on specific proteins or markers found on cancer cells, while aiming to reduce the impact on healthy cells.  

Tagraxofusp (Elzonris)

The first FDA-approved targeted therapy for BPDCN was tagraxofusp (Elzonris, Stemline), a CD123-directed cytotoxin. Elzonris works by binding to CD123, a protein that is overexpressed on BPDCN cells, and delivering a toxin that eliminates the cells. 

Read more about tagraxofusp for BPDCN

Pivekimab sunirine (PVEK)

Pivekimab sunirine (PVEK, Abbvie) is an antibody-drug conjugate currently being studied for the treatment of BPDCN. It works by attaching to the CD123 marker on BPDCN cells and delivering a targeted treatment to those cells while limiting impact on healthy tissue.

In 2020, the U.S. Food and Drug Administration (FDA) granted pivekimab sunirine, also known as PVEK (AbbVie), breakthrough therapy status, which sped up its review as a treatment for relapsed/refractory BPDCN.

Read more about PVEK for BPDCN

Azacitidine

Azacitidine is a type of drug called a hypomethylating agent. It is often used to treat AML and MDS. It works by helping to stop the growth of cancer cells and supports the production of normal blood cells in bone marrow.   

Read more about azacitidine

Venetoclax (Venclexta)

Venetoclax (Venclexta) is a BCL-inhibitor used to treat several types of leukemia. It works by blocking the BCL-2 protein, causing the destruction of cancerous cells. 

Venetoclax is not currently FDA-approved for the treatment of BPDCN. However, in some cases, because of similarities to types of leukemia like AML, doctors often prescribe venetoclax-based treatments to treat BPDCN. 

Ongoing research is exploring the benefit of combining tagraxofusp, venetoclax, and azacitidine for the treatment of BPDCN. 

Read more about venetoclax