Avatrombopag Receives FDA Approval for Treamtent of Pediatric Patients With Chronic Immune Thrompcytopenia

Avatrombopag (Doptelet; Sobi) has been approved by the FDA for the treatment of pediatric patients aged 1 year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a prior therapy. The decision is based on clinically meaningful data from the AVA-PED-301 study (NCT04516967).¹

3D rendering of platelets | Image Credit: © Corona Borealis - stock.adobe.com

Thrombocytopenia refers to an autoimmune condition characterized by low platelet counts, leading to excessive bruising, heavier bleeding from minor cuts, blood in the urine or stool, or unusually heavy menstrual cycles. ITP refers to newly diagnosed, persistent, or chronic disease duration that is typically diagnosed within 3 months of symptom onset. ITP occurs in 3% to 10% of children with 1 or more comorbidities but is often acute and resolves spontaneously. Despite efficacious treatments that lead to remission, many patients relapse after multiple lines of therapy.^2-4

Avatrombopag is an oral drug that mimics thrombopoietin, a natural compound that stimulates the production of platelets. It was initially approved in 2018 for thrombocytopenia in adults with chronic liver disease scheduled to undergo a procedure. In 2019, the FDA approved avatrombopag’s expanded indication for treatment of adults with chronic immune thrombocytopenia (ITP) that hasn't responded satisfactorily to a previous therapy.^2,5,6

“Since its introduction in 2019, [avatrombopag] has been a cornerstone therapy for chronic ITP in adults,” Duane Barnes, president of Sobi North America, told Yahoo Finance. “This approval not only reinforces our commitment to innovation but also allows us to expand the treatment experience for patients and their families by offering [avatrombopag] in 2 formulations.”²

The approval of avatrombopag for treatment of pediatric patients with ITP was supported by data from AVA-PED-301, a global, randomized, phase 3 study evaluating the efficacy, safety, and pharmacokinetics of the agent. The trial investigators randomized patients 3:1 to receive avatrombopag or placebo administered orally once daily for 12 weeks. The primary outcome measured was durable platelet response, with an alternative primary end point of platelet response.¹

The investigators reported that 27.8% of patients in the avatrombopag arm achieved the study’s primary end point of durable platelet response compared with 0% in the placebo arm (P = .0077; 95% CI, 15.8–39.7).²

A platelet response, the alternative primary endpoint, was achieved by 81.5% of patients receiving avatrombopag, compared with none in the placebo group (P < .0001; 95% CI, 71.1–91.8). By day 8, 55.6% of avatrombopag-treated patients had a platelet count of 50×10⁹/L or more without the use of rescue therapy (95% CI, 41.4%–69.1%; P < .0001), while no patients in the placebo group met this threshold (95% CI, 0.0%–16.1%).²

Avatrombopag was generally well-tolerated. In pediatric patients with persistent or chronic ITP, the most common adverse reactions (10% or more) included viral infection, nasopharyngitis, cough, pyrexia, and oropharyngeal pain.²

“[Avatrombopag] represents a significant advancement in the treatment of children and adolescents with persistent or chronic ITP,” said Rachael Grace, MD, MMSc, pediatric hematologist and director of Hematology Clinical Research at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, and lead investigator of the AVA-PED-301 study, in coverage from Yahoo Finance. “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 [avatrombopag] for pediatric ITP offers families a new treatment option that can help address challenges in managing ITP in pediatric patients.”²

REFERENCES

1. Avatrombopag for the treatment of thrombocytopenia in pediatric subjects with immune thrombocytopenia for ≥6 months. Updated January 17, 2025. Accessed July 25, 2025. https://clinicaltrials.gov/study/NCT04516967

2. Sobi announces U.S. 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). July 25, 2025. Accessed July 25, 2025. https://finance.yahoo.com/news/sobi-announces-u-food-drug-110000071.html

3. Thrombocytopenia (low platelet count). Mayo Clinic. May 13, 2025. Accessed July 25, 2025. https://www.mayoclinic.org/diseases-conditions/thrombocytopenia/symptoms-causes/syc-20378293

4. Min Lee J. Advances in management of pediatric chronic immune thrombocytopenia: a narrative review. J Yeungnam Med Sci. January 9, 2023. doi: 10.12701/jyms.2022.00745

5. Avatrombopag (oral route). Mayo Clinic. July 1, 2025. Accessed July 25, 2025. https://www.mayoclinic.org/drugs-supplements/avatrombopag-oral-route/description/drg-20443767

6. FDA approves avatrombopag for thrombocytopenia in adults with chronic liver disease. FDA. May 21, 2018. Accessed July 25, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-avatrombopag-thrombocytopenia-adults-chronic-liver-disease

7. FDA expands Avatrombopag’s indication for chronic immune thrombocytopenia. ASH Clinical News. June 27, 2019. Accessed July 25, 2025. https://ashpublications.org/ashclinicalnews/news/4592/FDA-Expands-Avatrombopag-s-Indication-for-Chronic