FDA

Doptelet approved for immune thrombocytopenia in children

July 30, 2025

FDA approved Doptelet (avatrombopag) for the treatment of thrombocytopenia in pediatric patients ≥1 year of age with persistent or chronic immune thrombocytopenia (ITP) who’ve had an insufficient response to a prior therapy. The approval also includes a new formulation, Doptelet Sprinkle (avatrombopag) oral granules, for use in children ages 1 to <6 years.

Efficacy

The pediatric approval was based on data from the randomized phase 3 AVA-PED-301 trial (NCT04516967) that evaluated the efficacy, safety, and pharmacokinetics of Doptelet in the treatment of pediatric patients with ITP. Results showed that 27.8% of Doptelet recipients achieved the primary endpoint of durable platelet response* vs. 0% of placebo patients in the absence of rescue medication (p=0.0077; 95% confidence interval [CI], 15.8-39.7). In addition, 81.5% of Doptelet recipients achieved the alternative primary endpoint of platelet response† vs. no patients in the placebo group (p=<0.0001; 95% CI 71.1-91.8). At Day 8, 55.6% of Doptelet recipients (95% CI, 41.4%, 69.1%) had a platelet count ≥50×10⁹/L in the absence of rescue therapy (p<0.0001) vs. no placebo patients (95% CI, 0.0%, 16.1%).

Safety

The most common adverse reactions (≥10%) in the trial were viral infection, nasopharyngitis, cough, pyrexia, and oropharyngeal pain.

* Durable response was defined as the proportion of patients achieving a platelet count ≥50×10⁹/L for six of the last eight weeks in the 12-week core phase of the AVA-PED-301 study.

† Platelet response was defined as the proportion of participants achieving ≥2 consecutive platelet assessments ≥50×10⁹/L in the core phase in the absence of rescue medication.

Sources:

Doptelet (avatrombopag) [package insert]. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/210238s010lbl.pdf Revised July 2025. Accessed July 29, 2025.

Sobi announces US Food and Drug Administration approves Doptelet® (avatrombopag) for the treatment of thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia (ITP) [News release]. 2025. https://www.sobi.com/usa/en/news-releases/sobi-announces-us-food-and-drug-administration-approves-dopteletr-avatrombopag-treatment-thrombocytopenia-pediatric-patients-one-year