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The Food and Drug Administration has approved tagraxofusp-erzs (Elzonris) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients, 2 years of age and older.
Approval was based on efficacy in two cohorts of patients in a single-arm clinical trial. Seven patients (54%) out of 13 with untreated BPDCN achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc) in the first cohort. In the second cohort of 15 patients, one patient achieved CR and one patient achieved CRc.
Common side effects for patients receiving tagraxofusp-erzs infusion were capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia, chills, and weight increase. Most common laboratory abnormalities were decreases in lymphocytes, albumin, platelets, hemoglobin, and calcium, and increases in glucose and liver enzymes (ALT and AST), the FDA said in a press statement.
The FDA placed a Boxed Warning on the drug to alert health care professionals and patients about the increased risk of capillary leak syndrome and recommends that health care providers monitor liver enzyme levels for signs of intolerance to the infusion.
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 FDA said.
“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,” Richard Pazdur, MD, 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, said in the statement.
The Food and Drug Administration has approved tagraxofusp-erzs (Elzonris) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients, 2 years of age and older.
Approval was based on efficacy in two cohorts of patients in a single-arm clinical trial. Seven patients (54%) out of 13 with untreated BPDCN achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc) in the first cohort. In the second cohort of 15 patients, one patient achieved CR and one patient achieved CRc.
Common side effects for patients receiving tagraxofusp-erzs infusion were capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia, chills, and weight increase. Most common laboratory abnormalities were decreases in lymphocytes, albumin, platelets, hemoglobin, and calcium, and increases in glucose and liver enzymes (ALT and AST), the FDA said in a press statement.
The FDA placed a Boxed Warning on the drug to alert health care professionals and patients about the increased risk of capillary leak syndrome and recommends that health care providers monitor liver enzyme levels for signs of intolerance to the infusion.
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 FDA said.
“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,” Richard Pazdur, MD, 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, said in the statement.
The Food and Drug Administration has approved tagraxofusp-erzs (Elzonris) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients, 2 years of age and older.
Approval was based on efficacy in two cohorts of patients in a single-arm clinical trial. Seven patients (54%) out of 13 with untreated BPDCN achieved complete remission (CR) or CR with a skin abnormality not indicative of active disease (CRc) in the first cohort. In the second cohort of 15 patients, one patient achieved CR and one patient achieved CRc.
Common side effects for patients receiving tagraxofusp-erzs infusion were capillary leak syndrome, nausea, fatigue, peripheral edema, pyrexia, chills, and weight increase. Most common laboratory abnormalities were decreases in lymphocytes, albumin, platelets, hemoglobin, and calcium, and increases in glucose and liver enzymes (ALT and AST), the FDA said in a press statement.
The FDA placed a Boxed Warning on the drug to alert health care professionals and patients about the increased risk of capillary leak syndrome and recommends that health care providers monitor liver enzyme levels for signs of intolerance to the infusion.
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 FDA said.
“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,” Richard Pazdur, MD, 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, said in the statement.