FDA Approves Nplate® (Romiplostim) For Use In Pediatric Patients With Immune Thrombocytopenia
"Today's approval underscores our long-standing commitment to making a positive impact on the lives of patients with rare and difficult-to-treat hematological disorders," said
The approval was based on two placebo-controlled studies – Phase 3 and Phase 1/2 – evaluating the safety and efficacy of Nplate in pediatric patients. In the Phase 3 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
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.1 The treatment goal for children with ITP is to achieve and maintain a platelet count that reduces the risk of bleeding.2
About Nplate® (romiplostim)
Nplate is a thrombopoietin (TPO) receptor agonist that mimics the body's natural TPO and is designed to increase platelet counts in patients with chronic immune thrombocytopenia (ITP).3 In the
Nplate is also approved in 67 countries, including
For more information about Nplate, please visit www.Nplate.com.
Important U.S. Nplate® Safety Information
Risk of Progression of Myelodysplastic Syndromes to Acute Myelogenous Leukemia
- In Nplate® clinical trials of adult patients with myelodysplastic syndromes (MDS) and severe thrombocytopenia, progression from MDS to acute myelogenous leukemia (AML) has been observed.
- Nplate® is not indicated for the treatment of thrombocytopenia due to MDS or any cause of thrombocytopenia other than chronic ITP.
- Thrombotic/thromboembolic complications may result from increases in platelet counts with Nplate® use. Portal vein thrombosis has been reported in patients with chronic liver disease receiving Nplate®.
- To minimize the risk for thrombotic/thromboembolic complications, do not use Nplate® in an attempt to normalize platelet counts. Follow the dose adjustment guidelines to achieve and maintain a platelet count of ≥ 50 x 109/L.
Loss of Response to Nplate®
- Hyporesponsiveness or failure to maintain a platelet response with Nplate® should prompt a search for causative factors, including neutralizing antibodies to Nplate®.
- To detect antibody formation, submit blood samples to
Amgen(1-800-772-6436). Amgenwill assay these samples for antibodies to Nplate® and thrombopoietin (TPO).
- Discontinue Nplate® if the platelet count does not increase to a level sufficient to avoid clinically important bleeding after 4 weeks at the highest weekly dose of 10 mcg/kg.
- In the placebo-controlled trials of adult ITP patients, headache was the most commonly reported adverse drug reaction, occurring in 35% of patients receiving Nplate® and 32% of patients receiving placebo. Headaches were usually of mild or moderate severity.
- Most common adverse reactions in adults (≥ 5% higher patient incidence in Nplate® versus placebo) were Arthralgia (26%, 20%), Dizziness (17%, 0%), Insomnia (16%, 7%), Myalgia (14%, 2%), Pain in Extremity (13%, 5%), Abdominal Pain (11%, 0%), Shoulder Pain (8%, 0%), Dyspepsia (7%, 0%), and Paresthesia (6%, 0%).
- In pediatric patients of age > 1 year receiving romiplostim for ITP, adverse reactions with an incidence of > 25% in the two randomized trials were: contusion (41%), upper respiratory tract infection (31%), and oropharyngeal pain (25%).
- Most common adverse reactions (≥ 5% incidence and > 5% more frequent in the romiplostim arm) across the two placebo-controlled trials were contusion (41%), upper respiratory tract infection (31%), oropharyngeal pain (25%), pyrexia (24%), diarrhea (20%), rash (15%), and upper abdominal pain (14%).
Nplate® administration may increase the risk for development or progression of reticulin fiber formation within the bone marrow. This formation may improve upon discontinuation of Nplate®. In a clinical trial, one patient with ITP and hemolytic anemia developed marrow fibrosis with collagen during Nplate® therapy.
Women who become pregnant during Nplate® treatment are encouraged to enroll in
Please see full U.S. Prescribing Information and Medication Guide at www.Nplate.com.
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National Organization for Rare Disorders. Immune Thrombocytopenia. https://rarediseases.org/rare-diseases/immune-thrombocytopenia/. Accessed Nov. 2, 2018. Children's National Health System. Pediatric Idiopathic Thrombocytopenia Purpura (ITP). https://childrensnational.org/choose-childrens/conditions-and-treatments/blood-marrow/idiopathic-thrombocytopenia-purpura-itp. Accessed Nov. 2, 2018
- Nplate® (romiplostim) prescribing information,
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