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

Stemline Therapeutics Inc's Elzonris has recently been approved by the U.S. Food and Drug Administration (FDA) 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, told.

Elzonris will be commercially available in early 2019, the company said.

The labeling for Elzonris carries FDA's harshest boxed warning that flags increased risk of capillary leak syndrome, which may be life-threatening or fatal to patients in treatment.

Last year, the drug developer said a patient death had occurred in the clinical studies of Elzonris, after developing capillary leak syndrome, a "well-documented side effect" of the drug.

"Boxed warning should have been expected given the history of capillary leak syndrome, but we don't think it will affect uptake," said David Nierengarten, Wedbush Securities analyst.

Stemline Therapeutics shares dropped as much as 9.9 percent to $7.82, before partially recovering to $8.5.

An aggressive and rare disease of the bone marrow and blood, BPDCN can affect multiple organs, including the lymph nodes and the skin, the FDA stated.

As per Roth Capital analyst Jotin Marango’s estimate, there are 350 BPDCN patients in the U.S., whereas the annual treatment cost of a patient is estimated at $285,000. The approval unlocks lateral indications for the drug, Marango said in a note.

Elzonris is also being tested for other indications, such as bone marrow disorders chronic myelomonocytic leukemia and myelofibrosis.

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