FDA News

FDA approves romiplostim for children with immune thrombocytopenia

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.

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.