The U.S. Food and Drug Administration (FDA) has approved Doptelet (avatrombopag) for the treatment of persistent or chronic immune thrombocytopenia (ITP) in patients one year and older who have had insufficient response to prior therapy.
ITP is a rare bleeding disorder characterized by too few platelets in the blood. This is caused by the destruction of platelets by the immune system. Symptoms may include bruising, nosebleed or bleeding in the mouth, bleeding into the skin, and abnormally heavy menstruation.
Avatrombopag is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics biologic effects of TPO in stimulating the development and maturation of megakaryocytes, increasing platelet count. It was approved by the FDA for the treatment of adults with ITP in 2019. The recent approval also includes a new formulation, Doptelet Sprinkle oral granules, for use in children ages one to less than six years, while the tablet is approved for those ages six years and older.
The approval follows results from the AVA-PED-301 clinical trial, a global, randomized, phase 3 study evaluating the safety, efficacy, and pharmacokinetics of avatrombopag in pediatric patients with ITP. The primary endpoint of durable platelet response was achieved in 27.8% of patients on avatrombopag versus 0% in the placebo group. Additionally, 85% of patients on avatrombopag achieved the alternative primary endpoint of platelet response versus 0% in the placebo group. At day 8, 55.6% of patients on treatment had a platelet count ≥50×10^9/L in the absence of rescue therapy versus no placebo patients.
Avatrombopag was generally well tolerated. The most common adverse reactions included viral infection, nasopharyngitis, cough, pyrexia, and oropharyngeal pain.
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To learn more about ITP and other rare hematologic conditions, visit https://checkrare.com/diseases/hematologic-disorders/
