FDA approves sBLA for NPlate to treat pediatric patients one year of age and older with immune thrombocytopenia. Amgen.

Amgen announced that the FDA has approved the supplemental Biologics License Application (sBLA) for Nplate (romiplostim) for the treatment of pediatric patients one year of age and older with immune thrombocytopenia (ITP) for at least six months who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy.

The approval was based on two placebo-controlled studies - Phase III and Phase 1/II - evaluating the safety and efficacy of Nplate in pediatric patients. In the Phase III study, published in The Lancet, rates of overall platelet response were increased with the Nplate group (71 percent) compared with placebo (20 percent), p<0.05. additionally, durable platelet response occurred more frequently with nplate (52 percent) compared with placebo (10 percent), p><0.05. in the two placebo-controlled trials, adverse reactions with an incidence of> 25 percent in the Nplate arm were contusion, upper respiratory tract infection and oropharyngeal pain. "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," said Michael D. Tarantino , M.D., president of the Bleeding and Clotting Disorders Institute and professor of Pediatrics and Medicine, University of Illinois College of Medicine-Peoria , Peoria, Ill. "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."

ITP is a rare, serious autoimmune disease characterized by low platelet counts in the blood (a condition known as thrombocytopenia) and impaired platelet production.1 In the U.S., the estimated prevalence of ITP in children is 5.3 per 100,000 children annually. The treatment goal for children with ITP is to achieve and maintain a platelet count that reduces the risk of bleeding.