The FDA granted breakthrough-therapy designation for firstline eltrombopag to treat severe aplastic anemia (SAA).
The thrombopoietin receptor agonist received this designation based on research conducted by the National Heart, Lung, and Blood Institute. The prospective, single-center, phase II trial showed that 52 percent of patients with treatment-naïve SAA who received eltrombopag in combination with standard immunosuppressive therapy achieved complete response at six months. The overall response rate (ORR) was 85 percent.
The most common AEs associated with eltrombopag were nausea, fatigue, cough, diarrhea, headache, shortness of breath, fever, dizziness, abdominal pain, muscle spasms, abnormal liver function tests, runny nose, or pain in the legs, arms, feet, or hands.
The drug is already approved as secondline therapy for refractory SAA, as well as for adults and children with chronic immune thrombocytopenia.
Sources: Novartis press release, January 4, 2018; Reuters, January 4, 2018.