Promacta Earns Breakthrough Therapy Designation by FDA for Severe Aplastic Anemia

Recently, the FDA granted a breakthrough therapy designation to eltrombopag (Promacta, Novartis) for combination use with standard immunosuppressive therapy as a first-line treatment for patients with severe aplastic anemia (SAA).

SAA is a rare blood disorder in which a patient’s bone marrow fails to produce enough red blood cells, white blood cells, and platelets.

Breakthrough designation therapy provides expedited FDA review for drugs that treat serious or life-threatening conditions that demonstrate a substantial improvement over existing therapies. Eltrombopag received this designation based on results from a phase 1/2 study conducted by researchers at the National Heart, Lung, and Blood Institute of the National Institutes of Health.

Novartis, who sponsors the drug, conducted an analysis of the study data and found that 52% of newly treated patients with SAA saw a complete response at 6 months when treated with eltrombopag at the start of and in conjunction with standard immunosuppressive treatment. The overall response rate was 85%.

“Promacta is a promising medicine that, if approved for first-line use in severe aplastic anemia, may redefine the standard of care for patients with this rare and seirous bone marrow condition,” said Samit Hirawat, MD, head of Novartis oncology global drug development in a statement. “We will continue to work closely with the FDA to make Promacta available to patients with SAA who are new to treatment as soon as possible.”

Eltrombopag is the only thrombopoietin receptor agonist indicated for the treatment of patients with SAA. Promacta, marketed as Revolade in most countries outside the United States, is also approved as a second-line therapy in patients with refractory SAA, and indicated for usage in adults and children with refractory chronic immune thrombocytopenia.