U.S. FDA approves Stemline Therapeutics’ rare blood disease treatment

(Reuters) – The U.S. Food and Drug Administration on Friday approved Stemline Therapeutics Inc’s Elzonris for the treatment of a rare blood disease in adults and children aged two years and above.

This is the first approved treatment for the condition, blastic plasmacytoid dendritic cell neoplasm (BPDCN), Richard Pazdur, director of the FDA’s Oncology Center of Excellence, said.

“The standard of care has been intensive chemotherapy, followed by bone marrow transplantation. Many patients with BPDCN are unable to tolerate this intensive therapy, so there is an urgent need for alternative treatment options.”

BPDCN is an aggressive and rare disease of the bone marrow and blood that can affect multiple organs, including the lymph nodes and the skin, the FDA said.

The condition is more common in men than women, and in patients who are 60 years and above.

The labeling for Elzonris contains a boxed warning, FDA’s harshest, flagging increased risk of capillary leak syndrome, which may be life-threatening or fatal to patients in treatment.

Stemline Therapeutics shares fell as much as 7.8 percent to $8 in early trading.