FDA grants Breakthrough status for Novartis’ Promacta

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The US FDA has granted Breakthrough Therapy designation to Novartis for Promacta in combination with standard immunosuppressive therapy for the treatment of patients with severe aplastic anaemia (SAA) as a first-line therapy.

Promacta – marketed as Revolade in most countries outside the US – is already approved as a second-line therapy in the refractory setting in SAA.

It is also approved for adults and children with chronic immune thrombocytopenia (ITP), for patients who are refractory to other treatments.

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. As a result, people living with this serious disease may experience debilitating symptoms and complications.

Up to one-third of patients do not respond to current therapies or relapse, causing symptoms to return.

“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, MD, Head, Novartis Oncology Global Drug Development.