Menarini Receives European Commission Approval of ELZONRIS (tagraxofusp), for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) - Seite 2

About ELZONRIS in the USA

ELZONRIS (tagraxofusp), a targeted therapy directed to CD123, is approved by the U.S. Food and Drug Administration (FDA) and commercially available in the U.S. for the treatment of adult and pediatric patients, two years or older, with BPDCN. For full prescribing information in the U.S., visit www.ELZONRIS.com.

ELZONRIS is also being evaluated in additional clinical trials in other indications, including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), acute myeloid leukemia (AML), and others are planned.

About BPDCN

BPDCN, formerly blastic NK-cell lymphoma, is an aggressive hematologic malignancy, often with cutaneous manifestations, with historically poor outcomes. BPDCN typically presents in the bone marrow and/or skin and may also involve lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. The World Health Organization (WHO) termed this disease "BPDCN" in 2008; previous names included blastic NK cell lymphoma and agranular CD4+/CD56+ hematodermic neoplasm. For more information, please visit the BPDCN disease awareness website at www.bpdcninfo.com.

About CD123

CD123 is a cell surface target expressed on a wide range of malignancies including blastic plasmacytoid dendritic cell neoplasm (BPDCN), certain myeloproliferative neoplasms (MPNs) including chronic myelomonocytic leukemia (CMML) and myelofibrosis (MF), acute myeloid leukemia (AML) (and potentially enriched in certain AML subsets), myelodysplastic syndrome (MDS), and chronic myeloid leukemia (CML). CD123 has also been reported on multiple myeloma (MM), acute lymphoid leukemia (ALL), hairy cell leukemia (HCL), Hodgkin's lymphoma (HL), and certain Non-Hodgkin's lymphomas (NHL). In addition, CD123+ cells have been detected in the tumor microenvironment of several solid tumors as well as in certain autoimmune disorders including cutaneous lupus and scleroderma.

Important Safety Information from EU SmPC

Warnings and precautions

• Capillary leak syndrome (CLS), including life-threatening and fatal cases have been reported with most events occurred during the first five days of the first cycle of treatment. Before initiating therapy, it should be ensured that patients have adequate cardiac function and serum albumin ≥ 3.2 g/dL. During treatment, serum albumin levels should be monitored prior to the initiation of each dose, or more often as clinically indicated. Additionally, patients should be assessed for other signs/symptoms of CLS. Patients should be made aware of identifying CLS symptoms and when to seek immediate medical attention. Intravenous albumin supplementation and dosing interruptions may be required. 
• Severe hypersensitivity reactions have been reported with ELZONRIS. 
• Thrombocytopenia and neutropenia have been reported in patients treated with ELZONRIS monotherapy.  The majority of events were reported in cycle 1 and cycle 2 of treatment, were not dose-limiting and did not recur in subsequent cycles.
• ELZONRIS can cause tumour lysis syndrome (TLS).
• Treatment with ELZONRIS has been associated with elevations in liver enzymes. Acute hepatic failure and liver encephalopathy has been reported in a patient treated with ELZONRIS at a higher dose (16 mcg/kg).

Summary of the Safety Profile