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First Treatment for Rare Bone Marrow and Blood Disease


On December 21, 2018, the FDA granted Stemline Therapeutics approval to market Elzonris (tagraxofusp-erzs) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients, two years of age and older.

 

BPDCN is an aggressive and rare disease of the bone marrow and blood that can affect multiple organs, including the lymph nodes and the skin. It often presents as leukemia or evolves into acute leukemia. The disease is more common in men than women and in patients 60 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, M.D., director of the 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.

 

The efficacy of Elzonris was studied in a clinical trial. Among patients with untreated BPDCN, 54 percent achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc). Among patients with relapsed or refractory BPDCN less than one percent of patients achieved CR or CRc.

 

Common side effects reported by patients in clinical trials were capillary leak syndrome (fluid and proteins leaking out of tiny blood vessels into surrounding tissues), nausea, fatigue, swelling of legs and hands (peripheral edema), fever (pyrexia), chills, and weight increase. Most common laboratory abnormalities were decreases in lymphocytes, albumin, platelets, hemoglobin, and calcium, as well as increases in glucose and liver enzymes (ALT and AST). Health care providers are advised to monitor liver enzyme levels and watch for signs of intolerance to the infusion. Women who are pregnant or breastfeeding should not take Elzonris because it may cause harm to a developing fetus or newborn baby.

 

The labeling for Elzonris contains a Boxed Warning to alert health care professionals and patients about the increased risk of capillary leak syndrome which may be life-threatening or fatal to patients in treatment.

 

See the FDA Announcement

 

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