PR87725

FLORENCE, Italy, January 21, 2021 /PRNewswire=KYODO JBN/ --

- 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 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 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 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 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

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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Source: Menarini I.F.R.