FDA greenlights Amgen’s Nplate for pediatric immune thrombocytopenia

Amgen’s Nplate (romiplostim) has received US Food and Drug Administration (FDA) approval for treating pediatric patients with immune thrombocytopenia (ITP). This latest approval extends the drug’s use to children aged one year and older.

The FDA’s endorsement of Nplate for pediatric ITP patients marks a significant milestone in treating this rare autoimmune disorder, characterized by low platelet counts and impaired platelet production. ITP poses serious health risks, including spontaneous bleeding and bruising, particularly in children who do not respond adequately to conventional treatments such as corticosteroids, immunoglobulins, or splenectomy.

Clinical Trial Successes

Amgen’s Nplate, a thrombopoietin (TPO) receptor agonist designed to stimulate platelet production, was approved based on data from two pivotal studies: a Phase 3 trial and a Phase 1/2 trial. These placebo-controlled studies demonstrated the drug’s efficacy and safety in pediatric patients with ITP.

In the Phase 3 clinical trial, 71% of patients treated with Nplate showed a significant platelet response compared to only 20% in the placebo group. Moreover, 52% of the Nplate cohort exhibited a durable platelet response, in contrast to just 10% in the placebo arm.

Safety Profile

The trials identified several adverse reactions with a frequency of more than 25% in the Nplate group. These included contusion, upper respiratory tract infection, and oropharyngeal pain. Despite these side effects, the overall benefits of Nplate for managing ITP in children appear to outweigh the risks.

Expert Commentary

Dr. Michael D. Tarantino, President of the Bleeding and Clotting Disorders Institute and Professor of Pediatrics and Medicine at the University of Illinois College of Medicine-Peoria, highlighted the significance of this approval. He noted, “Children with ITP are at risk for serious bleeding events and spontaneous bruising due to low platelet counts, which can be worrying for these young patients and their parents. Currently, these patients have a limited number of treatment options, especially for those with refractory disease. Today’s approval of Nplate offers new hope to the pediatric ITP community as it provides children with a new treatment option that may help to maintain safe platelet counts.”

Global Availability

Nplate, which mimics natural TPO to boost platelet counts, is already approved in 67 countries, including Australia, Canada, and Japan. This global recognition underscores its importance in treating ITP across diverse populations.

The FDA’s latest approval expands the therapeutic landscape for pediatric ITP patients, offering a promising new option for managing this challenging condition.