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The FDA grants the designation of innovative therapy for the treatment of rare blood disorder

FDA officials have awarded the designation of advanced therapy to eltrombopag (Promacta, Novartis) for first-line use, in combination with standard immunosuppressive therapy, in patients with severe aplastic anemia (SAA), the company said in a statement. .

Promacta has already been approved as second-line therapy in refractory environments in SAA, as well as for the treatment of adults and children with chronic immune thrombocytopenia (ITP) who are refractory to other treatments. It is marketed as Revolade in countries outside the United States.

According to research conducted by Heart, Lung and Blood Institute of the National Institutes of Health, the data demonstrated that 52% of patients with untreated SAA achieved a complete response in 6 months when treated with eltrombopag at baseline and at the same time as the standard immunosuppressive treatment, with an overall response rate of 85%.

Up to a third of patients do not respond to current therapies for AAS or relapse, causing symptoms that can be debilitating to return, according to Novartis.

"Promacta is a promising drug that, if approved for first-line use in severe aplastic anemia, can redefine the standard of care for patients with this index and severe bone marrow involvement." Samit Hirawat, MD, Director of Novartis Oncology Global Drug Development, said in the press release.

SAA is a rare blood disorder in which a patient's bone marrow can not produce enough red blood cells, white blood cells and platelets. Symptoms and complications such as fatigue, shortness of breath, recurrent infections and bruising or abnormal bleeding may occur.

The drug Novartis Promacta® receives the FDA Advanced Therapy designation for first-line use in severe aplastic anemia (ASA) [news release]. Novartis website. https://www.novartis.com/news/media-releases/novartis-drug-promactar-receives-fda-breakthrough-therapy-designation-first-line-use-severe-aplastic-anemia-saa. Retrieved on January 4, 2018.

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