Novel pair of therapies offers hope to patients with rare blood disorders

Two new therapies recently approved by FDA signal significant advancement in treating rare blood diseases.

The agency approved tagraxofusp-erzs (Elzonris, Stemline Therapeutics) infusion for blastic plasmacytoid dendritic cell neoplasm (BPDCN) for both adult and pediatric patients aged 2 years of and older.

“Prior to today’s approval…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 FDA’s Center for Drug Evaluation and Research, in an FDA statement.

Related: Top 3 new drugs to treat rare diseases

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, FDA said. It often presents as leukemia or evolves into acute leukemia.

The labeling for Elzonris contains a Boxed Warning to alert healthcare professionals and patients about the increased risk of capillary leak syndrome which may be life-threatening or fatal to patients in treatment.

FDA also cleared ravulizumab-cwvz (Ultomiris, Alexion Pharmaceuticals) injection to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disease.

Related: FDA clears new type of leukemia treatment

“The approval of Ultomiris will change the way that patients with PNH are treated,” Pazdur said. “Prior to this approval, the only approved therapy for PNH required treatment every 2 weeks, which can be burdensome for patients and their families. Ultomiris uses a novel formulation so patients only need treatment every 8 weeks, without compromising efficacy.”

The prescribing information for Ultomiris includes a Boxed Warning advising healthcare professionals and patients about the risk of life-threatening meningococcal infections and sepsis.

Read more: Major PBM’s formulary exclusions cause concerns