CONTENTS:
- A New Chapter in Pediatric ITP Treatment: FDA Approves Avatrombopag for Children Aged One and Up
- Watch Video: The Phases of ITP
A New Chapter in Pediatric ITP Treatment: FDA Approves Avatrombopag for Children Aged One and Up
Families navigating the challenges of pediatric immune thrombocytopenia (ITP) now have a new reason to feel hopeful. On July 25, 2025, the U.S. Food and Drug Administration (FDA) approved Doptelet® (avatrombopag) for children aged one and older with persistent or chronic immune thrombocytopenia (ITP) who have not responded adequately to previous treatments. This milestone expands access to a well-established therapy and introduces a new, child-friendly formulation designed to ease the burden of daily medication.
In children, ITP affects approximately five in 100,000 each year, and while many cases resolve on their own, up to 25% of newly diagnosed pediatric patients go on to develop chronic ITP. The condition can significantly impact quality of life, with symptoms ranging from fatigue and physical limitations to more serious bleeding complications.
Avatrombopag is a thrombopoietin receptor agonist (TPO-RA) that is currently indicated for the treatment of thrombocytopenia in adult patients with chronic liver disease scheduled for a procedure, and in adult patients with chronic ITP with insufficient response to prior treatment. The newly approved Doptelet Sprinkle formulation is indicated for children aged 1 to less than 6 years, while the Doptelet tablet formulation is approved for patients aged 6 years and older.
“Doptelet represents a significant advancement in the treatment of children and adolescents with persistent or chronic ITP,” said PDSA Medical Advisor Rachael Grace, MD, MMSc, Pediatric Hematologist and Director, Hematology Clinical Research at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, and lead investigator of the AVA-PED-301 study. “This therapy offers simple, flexible administration because it is oral, available as a tablet and now as a new pediatric sprinkle formulation and has no food restrictions. The approval of Doptelet for pediatric ITP offers families a new treatment option that can help address challenges in managing ITP in pediatric patients.”
The FDA’s decision was based on results from the AVA-PED-301 study, a global phase 3 clinical trial evaluating Doptelet’s efficacy and safety in children and adolescents with ITP. The study found that 27.8% of Doptelet-treated patients achieved a durable platelet response (defined as maintaining platelet counts above 50,000/uL for six of the last eight weeks of the study), compared to 0% in the placebo group. Additionally, 81.5% of patients exhibited a strong platelet response without requiring rescue medication, and by Day 8, more than half had achieved platelet counts above 50,000.
Doptelet was generally well-tolerated in the study. The most common side effects were mild and included viral infections, nasopharyngitis (common cold), cough, fever, and throat pain. As with other TPO-RAs, there is a small risk of thrombotic events, and Doptelet should not be used to normalize platelet counts. Families are encouraged to speak with their healthcare providers about the risks and benefits and to report any adverse reactions to the FDA. To learn more or report side effects, visit fda.gov/medwatch
www.sobi.com/usa/en/news-releases/sobi-announces-us-food-and-drug-administration-approves-dopteletr-avatrombopag-treatment-thrombocytopenia-pediatric-patients-one-year
Being diagnosed with ITP is scary—especially for kids. Learn more about PDSA's ITP POKE-R ClubSM and how this clinical support program can help your child cope with ITP.
