In a news release from March 2021, international pharmaceutical company the Menarini Group shared that it had joined a partnership with Nippon Shinyaku Co., Ltd. (“Nippon”) to develop and commercialize ELZONRIS (tagraxofusp) in Japan. Altogether, ELZONRIS is designed to treat patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). Although the journey to commercialization and development in Japan is still working, ELZONRIS is approved for BPDCN in both the United States and Europe.

ELZONRIS

Within Europe, ELZONRIS is used as a monotherapy for adults with BPDCN. Within the United States, the drug is indicated for the treatment of both pediatric and adult patients. This therapy targets CD123, which is expressed on BPDCN, acute myeloid leukemia (AML), chronic myeloid leukemia (CML), and other cancerous cells. The FDA further expands on ELZONRIS, explaining that:

ELZONRIS is injected into a vein (intravenous) by a healthcare provider once daily on days 1 through 5 of a 21-day cycle. It takes about 15 minutes to receive an ELZONRIS infusion, and patients are hospitalized for the duration of the first cycle.

Additionally, the FDA explains that ELZONRIS showed promise in clinical trials:

Out of 13 patients who had never been treated for BPDCN, 7 patients (54%) achieved no evidence of disease (complete remission or CR) or no evidence of disease with some skin changes not indicative of active disease (CRc) after treatment with ELZONRIS.

Out of 15 patients with worsening or resistant BPDCN, 2 patients experienced no evidence of disease (CR) or no evidence of disease with some skin changes not indicative of active disease (CRc) after treatment with ELZONRIS.

Thus, overall, ELZONRIS offers a new treatment option for patients in Japan with BPDCN. Hopefully, this will improve overall patient outcomes within this group. While ELZONRIS is relatively safe and well-tolerated, some side effects may occur. These include:

• Thrombocytopenia (low platelet count)
• Neutropenia (low neutrophil count)
• Anemia (low red blood cell count)
• Capillary leak syndrome (CLS)
• Hypersensitivity
• Nausea
• Fatigue
• Fever
• Tumor lysis syndrome (TLS)
• Increased liver enzyme levels

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

In the past, blastic plasmacytoid dendritic cell neoplasm (BPDCN) was once known as natural killer (NK) cell lymphoma. This rare and aggressive blood and bone marrow cancer easily metastasizes throughout the body. The condition was only standardized in 2008, so the medical field is still building an understanding of BPDCN. According to the Leukemia and Lymphoma Society (LLS):

Most often, BPDCN presents with features of both lymphoma and leukemia. There are little data about BPDCN and there is no established treatment.

As the continued research on BPDCN builds, there are some elements of the cancer we now know. For example, BPDCN affects males more than females. BPDCN is also often misdiagnosed. Common misdiagnoses include:

• Lupus
• Melanoma
• Acute myeloid leukemia (AML)

The average age of symptom onset usually occurs around 60-70 years old. Finally, BPDCN is characterized by deep purple skin lesions in around 80% of all cases. Other symptoms include:

• Fatigue
• Night sweats
• Fever
• Stomach pain
• Swollen lymph nodes
• Thrombocytopenia (low platelet count)
• Neutropenia (low white blood cell count)
• Anemia (low red blood cell count)