FDA approves first treatment for rare blood disease

FDA approved on Friday tagraxofusp-erzs (Elzonris—Stemline Therapeutics) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients, aged 2 years and older. "Prior to today's approval, there had been no FDA approved therapies for BPDCN.

FDA approved on Friday tagraxofusp-erzs (Elzonris—Stemline Therapeutics) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients, aged 2 years and older. "Prior to today's approval, there had been no FDA approved therapies for BPDCN. The standard of care has been intensive chemotherapy followed by bone marrow transplantation. Many patients with BPDCN are unable to tolerate this intensive therapy, so there is an urgent need for alternative treatment options," said Richard Pazdur, MD, director of FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. BPDCN, an aggressive and rare disease of the bone marrow and blood that can affect multiple organs, often presents as leukemia or evolves into acute leukemia. Adverse effects reported by patients in clinical trials include capillary leak syndrome, nausea, fatigue, swelling of legs and hands, fever), chills and weight increase. The drug's labeling includes a boxed warning to alert health care providers and patients about the increased risk of capillary leak syndrome, which may be life-threatening or fatal to patients in treatment.