55. Elzonris

fda
(FDA)

Active ingredient: tagraxofusp
Disease: blastic plasmacytoid dendritic cell neoplasm
Peak sales estimate: N/A
Approved: Dec. 21
Company: Stemline Therapeutics

The scoop: Patients suffering from the rare and aggressive blood disorder blastic plasmacytoid dendritic cell neoplasm (BPDCN) once had to go through intensive chemotherapy followed by bone marrow transplant. Not any more, thanks to an FDA approval for Stemline Therapeutics’ CD123-targeted therapy Elzonris. Because of the lack of alternative treatment, Elzonris was waved through on data from two small cohorts of patients who were either newly diagnosed or had relapsed on chemo. Even though Elzonris’ label carries a boxed warning over its risk of capillary leak syndrome, which led to patient deaths in the clinical study, H.C. Wainwright analyst Raghuram Selvaraju said physicians will prescribe it anyway, given that it’s the only drug approved specifically for BPDCN. The company now plans to launch the drug in early 2019, and it’s also testing it in other indications including chronic myelomonocytic leukemia, myelofibrosis and others. — Angus Liu

55. Elzonris

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