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U.S. Food and Drug Administration approval summary: Eltrombopag for the treatment of pediatric patients with chronic immune (idiopathic) thrombocytopenia
Author(s) -
Ehrlich Lori A.,
Kwitkowski Virginia E.,
Reaman Gregory,
Ko ChiaWen,
Nie Lei,
Pazdur Richard,
Farrell Ann T.
Publication year - 2017
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.26657
Subject(s) - medicine , eltrombopag , food and drug administration , immune thrombocytopenia , splenectomy , clinical trial , randomized controlled trial , pediatrics , drug , antibody , immunology , pharmacology , spleen
The U.S. Food and Drug Administration (FDA) approved eltrombopag for pediatric patients with chronic immune (idiopathic) thrombocytopenia (ITP) ages ≥6 on June 11, 2015, and ages ≥1 on August 24, 2015. Approval was based on the FDA review of two randomized trials that included 159 pediatric patients with chronic ITP who had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. This manuscript describes the basis for approval of these applications. The FDA concluded that eltrombopag has shown efficacy and a favorable benefit to risk profile for pediatric patients with chronic ITP.