FDA Approves First Treatment for Rare Blood Disease

On Dec. 21, 2018, FDA approved Elzonris (tagraxofusp-erzs) infusion for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients two years of age and older. Approval was granted to Stemline Therapeutics, a biopharmaceutical company focused on the development and potential commercialization of oncology therapeutics.

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. It often presents as leukemia or evolves into acute leukemia. The disease is more common in men than women and in patients 60 years and older, FDA reports.

The efficacy of Elzonris was studied in two cohorts of patients in a single-arm clinical trial. FDA granted this application breakthrough therapy and priority review designation. Elzonris also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

In November 2018, the European Medicines Agency granted the drug accelerated assessment for its upcoming centralized marketing authorization application, which Stemline expects to complete in the first quarter of 2019.

“Prior to today’s approval, there had been no FDA approved therapies for BPDCN. 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,” said Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in an agency press release.

Source: FDA, Stemline Therapeutics