Novartis anemia therapeutic is ready for first-line use in combination with standard immunotherapy.

Severe aplastic anemia (SAA) is a rare blood disorder indicated when a patient’s bone marrow loses its capacity to sufficiently produce enough platelets and white and red blood cells. Novartis announced it is another step closer to treating more patients with the disorder more effectively, now that the FDA has granted Breakthrough Therapy designation for the first-line use of Promacta® (eltrombopag), to treat people suffering from SAA in combination with standard immunotherapy.

Promacta, Novartis explained, is marketed as Revolade® in many countries outside the U.S. and is an approved second-line therapy for treating SAA in a refractory setting. The drug is also approved for children and adults with chronic immune thrombocytopenia (ITP) as well as people refractory to other treatments.

People with SAA, said Novartis, can experience a variety of debilitating symptoms and complications including excessive bruising, abnormal bleeding, troubled breathing and activity-limiting fatigue. As many as a third of all patients don’t respond to current therapies or relapse and experience the return of symptoms.

Samit Hirawat, Head of Novartis Oncology Global Drug Development, explained that 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." Hirawat further noted Novartis 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."

FDA grants Breakthrough Therapy to drugs intended to treat serious, life-threatening disease or disorders and able to demonstrate “a substantial improvement” over current standard therapies covering one or more “clinically significant” endpoints derived from preliminary clinical evidence.

Research conducted by the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) showed that when treated with Promacta at the initiation of and concurrently with standard immunosuppressive treatment, more than half (52%) of patients with treatment-naïve SAA achieved complete response at six months. According to Novartis, overall response was 85%.