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)

Core Insights - The FDA has approved Doptelet (avatrombopag) for treating thrombocytopenia in pediatric patients aged one year and older with persistent or chronic immune thrombocytopenia (ITP), including a new formulation, Doptelet Sprinkle, for children aged one to less than six years [1][2][3] Company Overview - Sobi North America, a subsidiary of Swedish Orphan Biovitrum AB (Sobi), is focused on providing innovative treatments for rare diseases, with Doptelet being a cornerstone therapy for chronic ITP since its introduction in 2019 [4][12] - The company aims to expand treatment options for patients and their families by offering Doptelet in both tablet and sprinkle formulations [4] Product Efficacy - The approval of Doptelet is based on the AVA-PED-301 study, which demonstrated that 27.8% of patients achieved a durable platelet response compared to 0% in the placebo group (p=0.0077) [3] - An alternative primary endpoint showed that 81.5% of Doptelet patients achieved a platelet response versus no patients in the placebo group (p<0.0001) [3] - At Day 8, 55.6% of Doptelet patients had a platelet count ≥50×10^9/L without rescue therapy, while no placebo patients achieved this [3] Safety Profile - Doptelet was generally well-tolerated, with common adverse reactions including viral infection, nasopharyngitis, cough, pyrexia, and oropharyngeal pain [3][7] - The product is associated with thrombotic and thromboembolic complications in specific patient populations, including those with chronic liver disease [5][6] Market Context - Immune thrombocytopenia (ITP) is a rare autoimmune disorder affecting approximately 100 people per million, with a significant incidence in children [4][10] - The approval of Doptelet for pediatric use addresses a critical need for effective treatment options in this demographic, as current therapies often lead to relapses [4][10]