Sobi begins avatrombopag phase 3 trial in immune thrombocytopenia in pediatrics
Swedish Orphan Biovitrum (Sobi) said that the first patient has been dosed with Doptelet (avatrombopag) in a phase 3 trial for the treatment of immune thrombocytopenia (ITP) in pediatric patients.
Avatrombopag is a thrombopoietin receptor agonist (TPO-RA).
Its phase 3 study – AVA-PED-301 will assess the efficacy, safety, and pharmacokinetics in the treatment of the autoimmune disorder that results when the body attacks its own platelets and destroys them too rapidly.
The late-stage study will feature 72 pediatric subjects (aged one or over to less than 18 years of age in nine countries. The participants in the AVA-PED-301 study will be randomly grouped to receive blinded therapy of either avatrombopag or placebo in a ratio of 3:1.
Ravi Rao – Head of Research and Development at Sobi said: “We are excited to announce the start of this important study.
“Our hope is to provide a new treatment option for children with ITP with the potential to decrease the treatment burden that impacts their daily lives.”
Doptelet mimics the biologic effects of TPO in triggering the development and maturation of megakaryocytes, which leads to higher platelet count.
It is approved by the US Food & Drug Administration (FDA) for the treatment of thrombocytopenia in adults having chronic liver disease who are scheduled to undergo a procedure.
Doptelet is also approved by the European Medicines Agency (EMA) for the treatment of severe thrombocytopenia in adults having chronic liver disease (CLD) who are scheduled to go through an invasive procedure.
Besides, Doptelet is approved by the two regulators for the treatment of primary chronic immune thrombocytopenia (ITP) in adults who are refractory to other treatments such as corticosteroids and immunoglobulins.