RELEASE: Menarini Group Announces ELZONRIS® Designated as an Orphan Drug for BPDCN

- Menarini Group announces ELZONRIS® (Tagraxofusp) designated as an orphan drug for BPDCN by the Ministry of Health, Labor and Welfare of Japan.

RELEASE: Menarini Group Announces ELZONRIS® Designated as an Orphan Drug for BPDCN

- Menarini Group announces ELZONRIS® (Tagraxofusp) designated as an orphan drug for BPDCN by the Ministry of Health, Labor and Welfare of Japan

FLORENCE, Italy and NEW YORK, Aug. 31, 2023 /PRNewswire/ -- Menarini Group ("Menarini"), a leading international pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. ("Stemline"), a subsidiary wholly owned by Menarini Group, today announced that Nippon Shinyaku Co., Ltd. (Nippon Shinyaku), Stemline's development partner in Japan, has received orphan drug designation for tagraxofusp from the Japanese Ministry of Health, Labor and Welfare (MHLW). ) for the expected indication of blastic plasmacytoid dendritic cell neoplasm (BPDCN).

BPDCN is an aggressive orphan hematologic malignancy with historically poor clinical outcomes. Stemline's ELZONRIS® (tagraxofusp) is the only approved treatment for patients with BPDCN, and the first and only CD123-targeted therapy approved, in both the United States and Europe.

Orphan drugs are designated by Japan's MHLW for treatments intended for use in fewer than 50,000 patients, where there is also particularly high medical need. Orphan drug designation can potentially shorten the period required to obtain regulatory approval in Japan by several months, thus allowing faster access for patients.

In Japan, Nippon Shinyaku is developing tagraxofusp, which is currently conducting a phase 1/2 clinical trial. In March 2021, Stemline and Nippon Shinyaku signed an exclusive license agreement for the development and commercialization of tagraxofusp in Japan, including the possibility of co-promotion.

"The MHLW's decision to grant orphan drug designation recognizes the potential positive impact that tagraxofusp could have on BPDCN patients in Japan, a patient population that currently has limited treatment options," said Elcin Barker Ergun, CEO of Menarini Group. "This achievement by our partner, Nippon Shinyaku, furthers our commitment to bringing transformative new therapeutic options to patients with difficult-to-treat cancers and delivering innovative medicines to people around the world."


BPDCN, formerly blastic NK-cell lymphoma, is an aggressive hematologic malignancy, often with cutaneous manifestations, with historically poor outcomes. BPDCN usually occurs in the bone marrow and/or skin and can also affect the lymph nodes and viscera. The BPDCN cell of origin is the precursor of plasmacytoid dendritic cells (pDC). 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) named this disease "BPDCN" in 2008; previous names included blastic NK-cell lymphoma and CD4/CD56 agranular hematodermal neoplasm. For more information, visit the BPDCN Disease Awareness website at

About the Menarini Group

Grupo Menarini is a leading international pharmaceutical and diagnostic company, with a turnover of more than 4.4 billion dollars and more than 17,000 employees. Menarini is focused on therapeutic areas with high unmet needs with products for cardiology, oncology, pulmonology, gastroenterology, infectious diseases, diabetology, inflammation and analgesia. With 18 production sites and 9 research and development centers, Menarini's products are available in 140 countries around the world. For more information, visit

About Stemline Therapeutics Inc.

Stemline Therapeutics, Inc. ("Stemline"), a wholly owned subsidiary of the Menarini Group, is a commercial stage biopharmaceutical company focused on the development and commercialization of novel cancer therapies. Stemline markets ORSERDU® (elacestrant) in the United States, an oral endocrine therapy indicated for the treatment of postmenopausal women or adult men who are estrogen receptor (ER) positive, human epidermal growth factor receptor 2 (HER2) negative, ESR1 mutated . Advanced or metastatic breast cancer with disease progression after at least one line of endocrine therapy. Stemline is also marketing ELZONRIS® (tagraxofusp-erzs), a novel CD123-targeted treatment for patients with blastic plasmacytoid dendritic cell neoplasia (BPDCN), an aggressive blood cancer, in the United States and Europe, which is the only approved treatment for BPDCN in US and EU to date. Stemline also markets NEXPOVIO®, an XPO1 inhibitor for multiple myeloma, in Europe. Stemline also has an extensive clinical portfolio of small molecules and biologics in various stages of development for a variety of solid and hematologic cancers.

Acerca de Nippon Shinyaku

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