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Sobi announces positive Phase 3 results

Sobi has announced results from the AVA-PED-301 study, evaluating the efficacy and safety of avatrombopag (Doptelet) for the treatment of paediatric patients with immune thrombocytopenia (ITP) of at least 6 months’ duration.

The study enrolled 75 subjects between 1 and <18 years and the primary endpoint, durable platelet response, was met in 28% of avatrombopag subjects in comparison with 0% of placebo subjects.

“Considering the challenges in treatment administration, coupled with variable and transient responses, frequent relapses, and associated toxicities from existing therapies, an unmet medical need currently exists in managing ITP among children and adolescents,” said Lydia Abad-Franch, Head of Research, Development, and Medical Affairs, and Chief Medical Officer at Sobi. “In line with Sobi’s mission to transform the lives of people with rare and debilitating diseases, we will continue to work towards enhancing care and addressing the distinctive challenges faced by younger ITP patients and their caregivers.”

The key secondary endpoint of two consecutive platelet counts ≥50×109/L was met in 81.5% of avatrombopag subjects in comparison with 0% of placebo subjects. A platelet response at Day 8 was observed in 56% of avatrombopag subjects and 0% of placebo subjects, while rescue therapy use occurred in 7% of avatrombopag subjects and 43% of placebo subjects.

Immune thrombocytopenia

Immune thrombocytopenia (ITP) is an autoimmune disorder characterised by low numbers of platelets, leading to bruising and an increased risk of bleeding. It is estimated that up to 100 people per million live with ITP. In children, ITP occurs in 5 out of 100,000 children per year1. Currently no cure is available, and patients usually relapse after various treatments yet still require treatment to reduce the risk of clinically significant bleeding.

Photo of Lydia Abad-Franch: Sobi