On July 25, 2025, the U.S. Food and Drug Administration (FDA) approved avatrombopag (Doptelet) for the treatment of thrombocytopenia in pediatric patients aged 1 year and older with persistent or chronic immune thrombocytopenic purpura (ITP) who have not responded adequately to prior treatment. This approval also includes a new dosage form—an oral powder—for children aged 1 to 6 years and younger.
“The approval of avatrombopag (Doptelet) is a significant advancement in the treatment of persistent or chronic ITP in children and adolescents aged 1 year and older,” said Rachael Grace, MD, MD, PhD, Pediatric Hematologist, Director of Clinical Research in Hematology at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, and principal investigator of the AVA-PED-301 study. “This therapy is simple and flexible in its administration: it is an oral formulation, available in tablet form, and now a pediatric-specific powder form, and can be taken without food restrictions. The approval of this new type of avatrombopag (Doptelet) for the treatment of pediatric ITP provides families with a new treatment option and helps address many challenges in the management of pediatric ITP.”
Overall, avatrombopag (Doptelet) is well tolerated. The most common adverse reactions (incidence ≥10%) in pediatric patients with persistent or chronic ITP include viral infection, nasopharyngitis, cough, fever, and oropharyngeal pain.
“Since its launch in 2019, avatrombopag (Doptelet) has been a cornerstone therapy for the treatment of chronic ITP in adults,” said Duane Barnes, President of Sobi North America. “This approval not only underscores our commitment to innovation but also further expands treatment options for patients and their families by offering avatrombopag (Doptelet) in two dosage forms.”
About Immune Thrombocytopenic Pulmonary Disease (ITP)
Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by a low platelet count, leading to increased bruising and bleeding risk. It is estimated that approximately 100 people per million population worldwide have ITP. In children, the annual incidence of ITP is 5 cases per 100,000 children; up to 25% of newly diagnosed pediatric ITP patients will develop chronic ITP. Currently, there is no cure for ITP, and patients often relapse after various treatments.
Avatrombopag (Doptelet) Indications
Avatrombopag (Doptelet) is a thrombopoietin receptor agonist approved for the following indications:
(1) Thrombocytopenia in adult patients with chronic liver disease scheduled for surgery;
(2) Thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an inadequate response to prior treatment;
(3) Thrombocytopenia in pediatric patients aged 1 year and older with persistent or chronic immune thrombocytopenia who have had an inadequate response to prior treatment.
