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

- ELZONRIS is the first approved treatment for patients with BPDCN, and the first approved CD123-targeted therapy, in Europe

- Approval is based on the results of the largest prospective clinical trial ever conducted in patients with treatment-naïve or previously-treated BPDCN

FLORENCE, Italy, Jan. 22, 2021 /PRNewswire/ -- The Menarini Group, a privately held Italian pharmaceutical and diagnostics company, announced today that the European Commission (EC) has granted a marketing authorization, for ELZONRIS (tagraxofusp) as monotherapy for the first-line treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare, aggressive hematologic malignancy with dismal outcomes. The EC decision follows the positive opinion received from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in November 2020, and is based on the results of the largest prospective clinical trial ever conducted in patients with treatment-naïve or previously-treated BPDCN.

ELZONRIS has been granted orphan designation in Europe, and is now the first approved treatment for patients with BPDCN, and the first approved CD123-targeted therapy in Europe to address this high unmet medical need.

"For the first time, patients living with BPDCN in Europe will have the possibility to benefit from a treatment tailored against this aggressive malignancy," commented Elcin Barker Ergun, CEO of the Menarini Group. "The approval of ELZONRIS can enable a significant change in the therapeutic approach for BPDCN, since it provides clinicians with a targeted therapy to help patients suffering from this terrible disease. We are working to make ELZONRIS available in Europe in the shortest time possible, as part of our commitment to deliver innovative and effective medicines for people affected by serious health conditions".

ELZONRIS is a targeted therapy directed to CD123, already approved by the FDA and marketed in the US since 2019 by Stemline Therapeutics, now part of the Menarini Group.

ELZONRIS was approved by the U.S. Food and Drug Administration (FDA) in 2018, where it is currently available for the treatment of BPDCN in adult and pediatric patients, two years or older.

About ELZONRIS^® in the European Union

ELZONRIS^® (tagraxofusp) is indicated as monotherapy for the first-line treatment of adult patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). ELZONRIS should be administered under the supervision of a physician experienced in the use of anti-cancer agents.

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

• The most serious adverse reaction that may occur during ELZONRIS treatment is CLS which was reported in 18% of patients with a median time to onset of CLS of 6 days.
• Adverse reactions occurring in ≥ 20% of patients treated with ELZONRIS were hypoalbuminemia, increased transaminases, thrombocytopenia, nausea, fatigue and pyrexia.
• Adverse reactions grade 3 and above according to the Common Terminology Criteria for Adverse events (CTCAE) and occurring in > 5% of patients were increased transaminases, thrombocytopenia and anaemia.

For full prescribing information in Europe please refer to the full SmPC at https://www.ema.europa.eu

About the Menarini Group

Menarini Group is a leading international pharmaceutical company with a presence in 140 countries, including a direct presence in over 70 countries. Its global platform extends throughout Europe, U.S., Central America, Africa, the Middle East and Asia Pacific, and generates over $4.2 billion in annual sales. Menarini is committed to oncology, with an already commercialized product in the US and several new investigational drugs in development for the treatment of a variety of tumors. For over 130 years, Menarini has also been investing in the development, production and distribution of pharmaceuticals to serve patients and physicians around the world with a full portfolio of products covering a number of different therapeutic areas.

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