The U.S. Food and Drug Administration (FDA) granted approval to a supplemental Biologics License Application for romiplostim that updates the drug’s label to include data supporting its earlier use in adult patients with immune thrombocytopenia (ITP). Earlier administration could potentially reduce the need for prolonged steroid use, according to the drug’s manufacturer.
The label changes were based on data from a phase II trial that included 75 adults with previously treated ITP who had been diagnosed within six months of enrollment. During the 12-month treatment period, the median duration of platelet response (defined as achieving ≥50×109/L) was 11 months (range not reported). The median time to first platelet response was 2.1 weeks after treatment start (range not reported). Twenty-four patients (32%) achieved remission lasting for at least six months (defined as maintaining a platelet count ≥50×109/L in the absence of any ITP medication).
Common adverse events, occurring in ≥5% of patients and in ≥5% more patients in the romiplostim-treated group than the placebo group, included bronchitis, sinusitis, vomiting, arthralgia, myalgia, headache, dizziness, diarrhea, upper respiratory tract infection, cough, nausea, and oropharyngeal pain.
Romiplostim has previously been approved for the treatment of adults with newly diagnosed or persistent ITP, as well as children younger than age 1 who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
Source: Amgen press release, October 18, 2019.