The Food and Drug Administration (FDA) has approved the supplemental Biologics License Application (sBLA) for Nplate (romiplostim; Amgen) to expand treatment to newly diagnosed adult patients with immune thrombocytopenia who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.
The approval was based on data from a phase 2, open-label, single-arm study that included 75 adult patients with immune thrombocytopenia diagnosed ≤6 months prior who had an insufficient response to first-line treatment (median time from diagnosis to study enrollment was 2.2 months). The primary end point was the median number of months with platelet response (≥50 x 109/L) during the 12-month treatment period, while a key secondary end point included the percentage of patients in remission, defined as maintaining a platelet count ≥50 x 109/L for at least 6 months in the absence of any therapy).
Results of the study showed that the median number of months with platelet response was 11 months (95% CI: 10, 11), while the median time to first response was 2.1 weeks (95% CI: 1.1, 3.0). Moreover, during the 12-month treatment period, 93% of patients achieved 1 or more platelet responses. In addition, 32% of patients achieved remission in the absence of Nplate and any other therapies to treat immune thrombocytopenia.
“These new data are the first of their kind to prospectively examine treatment-free remission as an outcome for patients with [immune thrombocytopenia],” said David M. Reese, MD, executive vice president of Research and Development at Amgen. “This approval will provide patients the opportunity to receive Nplate earlier in the course of their disease, potentially reducing their need for prolonged steroid use.”
For more information visit amgen.com.