Orphan Drug Status Granted for Novel Targeted Therapy to Treat Rare Hematologic Cancer


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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.

Blastic plasmacytoid dendritic cell neoplasm was previously known as blastic NK cell lymphoma and agranular CD4+/CD56+ hematodermic neoplasm. The disease most commonly affects middle-aged and older patients and is approximately three times more common in men than women. ■



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