According to a story from PR Newswire, the pharmaceutical and diagnostics company The Menarini Group recently announced that its drug tagraxofsup (marketed as Elzonris) has a received a positive opinion recommending approval from the Committee for Medicinal Products for Human Use (CHMP). This opinion from CHMP, which is part of the European Medicines Agency (EMA), recommends the use of this drug as a first line therapy for adult patients living with blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare disease.

About BPDCN

Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare blood disorder that affects plasmacytoid dendritic cells. It is projected that this disease comprises less than 0.5% of all blood disease cases. Plasmacytoid dendritic cells are a rare type of immune system cell that play a role in combating viral infections. The disease presents with characteristics similar to both leukemia and lymphoma. Although risk factors remain unknown, the BPDCN appears after mutations build up in plasmacytoid dendritic cell (pDC) precursors. Symptoms of the disease include ulcers, bruise-like patches, or skin lesions, swollen lymph nodes, liver, and/or spleen, anemia, and the presence of malignant pDCs throughout the body. Treatment often includes chemotherapy and stem cell transplant, but the disease almost always relapses after this treatment. Consequently, the five year survival rate for BPDCN is low. There is an urgent need for more effective therapies for this disease. To learn more about BPDCN, click here.

About Elzonris

With the urgent need for new treatments, the opinion will now be reviewed by the European Commission to determine eligibility for marketing authorization. Elzonris was approved for the treatment of this disease in the US in 2018 for patients age two or older. It is a therapy that targets CD123, and it is currently being tested in other CD123 positive malignancies, such as myelofibrosis, acute myeloid leukemia, and chronic myelomonocytic leukemia. It has the potential to be useful in other diseases where the CD123 target is present as well.

The opinion from CHMP was based on positive findings in the largest scale clinical trial evaluated BPDCN patients. The study included both previously treated patients and those who had not yet received any treatment.

With this opinion, an urgently needed therapy for BPDCN patients in the EU could soon gain approval.