FDA Grants Orphan Drug Status for Novel Targeted Therapy for the Treatment of Rare Hematologic Cancer
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to Stemline Therapeutics' SL-401 for the treatment of blastic plasmacytoid dendritic cell neoplasm, a rare and aggressive hematologic malignancy for which there is no effective treatment. SL-401 also has Orphan Drug status for the treatment of acute myeloid leukemia (AML).
SL-401 is a novel targeted therapy directed to the interleukin-3 receptor present on tumor bulk and cancer stem cells of multiple hematologic cancers. The agent has demonstrated single-agent clinical activity in patients with advanced hematologic cancers, including blastic plasmacytoid dendritic cell neoplasm, AML, and myelodysplastic syndrome.
About Blastic Plasmacytoid Dendritic Cell Neoplasm
Blastic plasmacytoid dendritic cell neoplasm (previously known as blastic NK cell lymphoma and agranular CD4+/CD56+ hematodermic neoplasm) is a rare and aggressive hematologic cancer for which there is no effective treatment. The disease most commonly affects middle-aged and older patients and is approximately three times more common in men than women.
Although blastic plasmacytoid dendritic cell neoplasm can be controlled for brief periods with standard chemotherapy, including high-dose chemotherapy with bone marrow transplantation, overall prognosis remains poor and the median overall survival from diagnosis is approximately 12 months. There are currently no approved therapies for the disease, and an optimal therapeutic regimen has not yet been established.
The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.
