FDA Grants Promacta Breakthrough Therapy Designation for Aplastic Anemia

This morning, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to Novatris’ eltrombopag (Promacta), a first-line treatment for severe aplastic anemia (SAA).

The designation was granted for the drug for use in combination with standard immunosuppressive therapy in treating the rare blood disorder. SAA is a condition in which the bone marrow does not produce enough blood cells for the body, and eltrombopag, a bone marrow stimulant, has previously received both FDA and European Union approval to treat adults and children with chronic immune thrombocytopenia (ITP) who are refractory to other treatments.

The investigative therapy was developed to imitate the thrombopoietin protein, and assist the body in the production of a greater number of platelets while improving poor cell counts.

The safety and efficacy of eltrombopag as a supplement to standard immunosuppressive therapy for patients with SAA were tested in phase 1 and phase 2 clinical trials sponsored by the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH). In the studies, over half (52%) of patients with treatment-naïve SAA achieved complete response at 6 months when treated with eltrombopag at baseline and along with standard horse antithymocyte globulin (h-ATG) and cyclosporine (CsA) therapies.

In the trials, overall response rate in patients 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 serious bone marrow condition,” said Samit Hirawat, Head, Novartis Oncology Global Drug Development. “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.”

The Breakthrough Therapy designation confirms eltrombopag’s potential to treat a serious or life-threatening disease or condition, and demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints based on preliminary clinical evidence.

Eltrombopag is currently approved in more than 45 countries for the treatment of patients with SAA, more than 100 countries for adult patients with chronic ITP, and more than 50 countries for the treatment of thrombocytopenia in patients with chronic hepatitis C.

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