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FDA approves romiplostim for children with immune thrombocytopenia

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The FDA today approved romiplostim for treatment of certain pediatric patients with immune thrombocytopenia, according to a press release from the agency.

The approval of romiplostim (Nplate, Amgen) applies to children aged 1 year or older who have had immune thrombocytopenia (ITP) for at least 6 months and had insufficient response to corticosteroids, immunoglobulins or splenectomy.

ITP is a common bleeding disorder in which blood does not clot as it should due to a low number of platelets or thrombocytes.

The FDA based the approval on results of two double-blind, placebo-controlled clinical trials, both of which included patients aged 1 year or older who had ITP for at least 6 months.

One study included children whose disease relapsed after or was refractory to at least one prior ITP therapy. Researchers randomly assigned these patients 2:1 to romiplostim (n = 42) or placebo (n = 20).

A higher percentage of patients assigned romiplostim achieved durable platelet response (52% vs. 10%; P < .05), defined as at least six weekly platelet counts of 50 × 10⁹/L or higher between week 18 and week 25 of treatment.

A higher percentage of romiplostim-treated patients also achieved overall platelet response (71% vs. 20%; P < .05), defined as durable or transient platelet response.

Patients in the romiplostim group achieved platelet counts of 50 x 10⁹/L or higher for a median 12 weeks, whereas patients assigned placebo attained that platelet count level for a median 1 week (P < .05).

The second study included patients diagnosed with ITP at least 6 months before enrollment.

Researchers randomly assigned patients 3:1 to romiplostim (n = 17) or placebo (n = 5).

Fifteen patients (88%) assigned romiplostim achieved a platelet count of 50 x 10⁹/L or higher for 2 consecutive weeks and an increase in platelet count of 20 × 10⁹/L or higher above baseline for 2 consecutive weeks during the treatment period. No patient assigned placebo achieved either of those thresholds.

The most common adverse reactions reported among at least 25% of pediatric patients treated with romiplostim include contusion, upper respiratory tract infection and oropharyngeal pain.

The FDA previously granted the agent orphan drug designation for this indication.