The US Food and Drug Administration (FDA) has accepted Sobi North America’s avatrombopag (DOPTELET) supplemental new drug application (sNDA) to treat paediatric immune thrombocytopenia.
The therapy is aimed at treating thrombocytopenia in children aged one and above, who are having persistent or chronic immune thrombocytopenia (ITP) and have not responded adequately to previous therapies.
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Sobi’s sNDA is supported by data from the AVA-PED-301 trial, which assessed the therapy’s safety and efficacy in treating young patients with ITP for a minimum of six months.
It met its primary endpoint, with a significant proportion of subjects achieving a durable platelet response during the 12-week treatment period.
The alternative primary efficacy endpoint was reached.
Avatrombopag, known under the brand name DOPTELET, is a thrombopoietin receptor agonist (TPO-RA) that does not compete with thrombopoietin (TPO) for receptor binding. It functions by stimulating the growth of megakaryocytes, which are bone marrow cells responsible for platelet production.
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By GlobalDataAn autoimmune disorder, ITP leads to lower platelet counts, causing an elevated bleeding risk. Symptoms vary and can range from bruising and rashes to severe bleeding and fatigue.
The US regulator also approved the company’s NDA for an oral suspension form of the therapy. This formulation allows the medication to be mixed with soft food or liquid.
Sobi North America chief medical officer Jamie Freedman said: “These application acceptances for avatrombopag brings us one step closer to much-needed, additional treatment options for adolescents and children with persistent and chronic ITP.
“Now, there’s an opportunity to address an unmet need in our paediatric population, with the bonus of an additional formulation, an oral suspension option that may be more suitable for some patients.”
Sobi North America is an affiliate of Swedish Orphan Biovitrum (Sobi), which entered an agreement to acquire CTI BioPharma for $1.7bn, in May 2023.
