Dive Brief:
- Promacta’s first FDA approval was for treatment of thrombocytopenia.
- The most recent approval is for the treatment of severe aplastic anemia (SAA) in patients who have not fully responded to immunosuppressive therapy (IST).
- The approval stems from an NIH-led, phase II trial in 43 patients with SAA. Patients involved in the study who had not responded to IST demonstrated a hematologic response to Promacta.
Dive Insight:
SAA is a disease in which the bone marrow does not make enough red blood cells, white blood cells, and platelets. Each year, there are 300 to 600 new cases diagnosed in the U.S. Roughly one-fourth to one-third of SAA patients do not respond to IST, and even among those who do respond, 30% to 40% relapse.
The outcomes can be bleak: About 40% of non-responders to IST die within five years of diagnosis either from infection or bleeding.
In January, Promacta was granted breakthrough therapy designation by the FDA, and in April it was given priority review. Promacta represents an important new treatment option for SAA and may significantly alter the prognosis for afflicted patients.