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FDA Approves Treatment for Thrombocytopenia
The FDA has approved romiplostim (Nplate; Amgen) for the treatment of immune thrombocytopenia (ITP) in children 1 year of age and older who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. According the FDA the treatment can be used for at least 6 months.
The drug approval was based on two double-blind placebo-controlled clinical trials in children 1 year and older with ITP for at least 6 months duration.
The first study included patients whose disease was refractory or relapsed after at least one prior ITP therapy. They were randomized (2:1) to receive Nplate (n=42) or placebo (n=20). According to the findings, durable platelet response was achieved in 22 patients (52%) who received Nplate and 2 patients (10%) in the placebo arm.
“Overall platelet response, defined as a durable or a transient platelet response, was achieved in 30 (71%) and 4 (20%) patients, respectively,” the FDA wrote in a press statement. “Patients who received romiplostim had platelet counts ≥ 50 x 109/L for a median of 12 weeks, compared to 1 week in patients who received placebo.”
In the second clinical trial, patients with ITP for at least 6 months prior to enrollment were randomized (3:1) to receive Nplate (n=17) or placebo (n=5). According to the findings of the second study, 15 patients who received Nplate achieved a platelet count ≥ 50 x 109/L for 2 consecutive weeks and an increase in platelet count of ≥ 20 × 109/L above baseline for 2 consecutive weeks during the treatment period (88%, 95% CI: 64%, 99%). Notably, no patient receiving placebo achieved either study endpoint.
The most common adverse reactions reported were contusion, upper respiratory tract infection, and oropharyngeal pain.—Julie Gould
