Country for PR: United Kingdom
Contributor: PR Newswire Europe
Friday, January 22 2021 - 05:25
Menarini Receives European Commission Approval of ELZONRIS (tagraxofusp), for the Treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
FLORENCE, Italy, January 21, 2021 /PRNewswire-AsiaNet/ --

- 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

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-naive 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(R) in the European Union

ELZONRIS(R) (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(R) in the USA

ELZONRIS(R) (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

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.


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

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 greater than or equal 
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 
- 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 greater than or equal 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

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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Source: Menarini I.F.R.