A Biologics License Application for ElzonrisTM (also known as tagraxofusp and SL-401) for the treatment of blastic plasmacytoid dendritic cell neoplasm has been accepted for filing by the United States FDA. In addition, the FDA has awarded Priority Review to this application with a target date of 21st February 2019, within the Prescription Drug User Fee Act. You can read the source press release here, at Stemline Therapeutics’s website.
About Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
BPDCN, previously known as natural killer cell leukaemia or lymphoma, is considered to be a form of acute myeloid leukaemia under WHO guidelines. According to the Leukemia and Lymphoma Society, the average age of diagnosis for this condition is between 60 and 70 years old, with more men than women receiving a diagnosis. It often presents features of both leukaemia and lymphoma. In approximately 80% of cases, the cancer affects the skin, however, as it progresses it may also involve the bone marrow and alter counts of white blood cells, red blood cells, and platelets. Currently, there is not an accepted standard of care for patients diagnosed with BPDCN.
About Biologics License Applications (BLA)
According to the FDA, the BLA is a request to the US FDA for permission to introduce, or deliver for introduction, a biological product into interstate commerce. In order to submit a BLA for FDA review, the product has to be associated with information, such as the applicant, product, and manufacturing information, pre-clinical and clinical studies, and labelling information.
Stemline Therapeutics’s BLA on behalf of ElzonrisTM has now been accepted for filing by the FDA.
About Priority Review
The FDA has also awarded Priority Review status to this application. This means that the FDA will attempt to review the application within six months, rather than the standard ten. Priority Review status is reserved for drugs or products that, if they were approved, would significantly improve treatment safety or effectiveness for patients with serious conditions. For more information about Priority Review, you can visit the FDA’s website here.
Elzonris is a drug that is designed to work by targeting a cell surface receptor called CD123 that is present on several malignancies. The drug has been investigated in a trial involving patients with BPDCN and is also being studied in clinical trials for the treatment of other conditions, including chronic myelomonocytic leukaemia and myelofibrosis. Elzonris has been awarded Orphan and Breakthrough Drug Designations.