Biopharmaceutical company Swedish Orphan Biovitrum AB (Sobi) (STO:SOBI) reported on Friday that the first patient has been dosed in a phase 3 study of Doptelet (avatrombopag) in children with immune thrombocytopenia (ITP).
ITP is an autoimmune disorder that occurs when the body attacks its own platelets and destroys them too quickly. In children with ITP, the body produces platelets normally but destroys them too quickly, with platelets surviving only a few hours instead of the normal 7-10 days. This results in a low platelet count in the blood and symptoms such as petechiae, easy bruising, and bleeding events. While spontaneous remission is common, 10-20% of children with the condition will develop chronic ITP.
Avatrombopag is an orally administered thrombopoietin receptor agonist (TPO-RA) that mimics the biologic effects of TPO in stimulating the development and maturation of megakaryocytes, resulting in increased platelet count.
The new phase 3 study will evaluate the efficacy, safety and pharmacokinetics of avatrombopag in paediatric subjects with ITP.
Avatrombopag is already approved by the US Food & Drug Administration (FDA) for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure, and by the European Medicines Agency (EMA) for the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure. It is also approved by the FDA and EMA for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).
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