Novartis ianalumab Phase III trial meets primary endpoint in immune thrombocytopenia

ITP is a rare autoimmune disorder characterized by low platelet counts leading to an increased risk of bleeding, bruising and chronic fatigue. Many people living with ITP cycle through multiple therapies, unable to achieve long-term disease control. There is a need for other treatment options with novel mechanisms of action that offer durable responses while reducing the burden of long-term treatment.

“For many people living with ITP, chronic treatment can disrupt their daily life due to the burden of regular dosing, dose adjustments and side effects,” said Shreeram Aradhye, M.D., President, Development and Chief Medical Officer, Novartis. “These positive top-line results from the Phase III study highlight the potential of ianalumab, if approved, to deliver long-term disease control with four once-monthly doses and enable extended time off treatment.”

Data is expected to be presented at an upcoming medical meeting and included in future regulatory submissions in 2027 along with results from the ongoing first-line ITP trial, VAYHIT1. Ianalumab has been granted Orphan Drug Designation by the US Food and Drug Administration and the European Medicines Agency. Recently, Novartis announced positive top-line results for ianalumab in adults with active Sjögren's disease.