Avatrombopag receives FDA approval to treat pediatric patients with chronic immune thrombocytopenia

Avatrombopag is approved by the U.S. Food and Drug Administration for the treatment of pediatric patients 1 year of age and older with persistent or chronic immune thrombocytopenia (ITP) who have had an inadequate response to prior therapy. The decision was based on clinically meaningful data from the AVA-PED-301 study (NCT04516967).

Thrombocytopenia refers to an autoimmune disorder characterized by a low platelet count that results in excessive bruising, worsening bleeding from minor cuts, blood in the urine or stool, or abnormally heavy menstrual cycles. ITP refers to a newly diagnosed, ongoing, or chronic condition, usually diagnosed within 3 months of symptom onset. ITP occurs in 3% to 10% of children with 1 or more comorbidities, but is usually acute and resolves spontaneously. Although effective treatments can lead to remission, many patients relapse after multiple lines of therapy.

Avatrombopag is an oral medication that mimics thrombopoietin, a natural compound that stimulates the production of platelets. It was originally approved in 2018 for the treatment of thrombocytopenia in adults with chronic liver disease who are scheduled to undergo surgery. In 2019, the U.S. Food and Drug Administration approved an expanded indication for avatropopag for the treatment of adults with chronic immune thrombocytopenia (ITP) who have not responded satisfactorily to previous treatments.

Since its launch in 2019, avatropopag has been the cornerstone therapy for adults with chronicITP. This approval not only strengthens the commitment to innovation but also enables the expansion of the treatment experience for patients and their families by offering [avatrombopag] in two formulations. Data from AVA-PED-301, a global randomized Phase 3 study evaluating the drug's efficacy, safety and pharmacokinetics, support approval of avatrombopag in children with ITP

Trial investigators randomly divided patients into two groups in a ratio of 3:1 to receive oral avatrombopag or placebo once daily for 12 weeks. The primary outcome measured was durable platelet response, as well as the other primary endpoint of platelet response. Researchers reported that 27.8% of patients in the avatropopag group achieved the study's primary endpoint of durable platelet response, compared with 0% in the placebo group (P=0.0077; 95% CI, 15.8-39.7).

Of the patients who received avatrombopag, 81.5% achieved a platelet response, the other primary endpoint, compared with none in the placebo group (P<0.0001; 95% confidence interval is 71.1-91.8). By day 8, 55.6% of avatropopag-treated patients had platelet counts of 50 × 10⁹/L or greater without the use of rescue therapy (95% CI, 41.4%-69.1%; P<0.0001), whereas no patients in the placebo group reached this threshold (95% CI, 0.0%-16.1%).

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

References:https://www.pharmacytimes.com/view/avatrombopag-receives-fda-approval-for-treamtent-of-pediatric-patients-with-chronic-immune-thrompcytopenia