Ianalumab reduced bleeding and depleted B cells in treatment-resistant ITP, while FDA approved gene therapy for Wiskott-Aldrich syndrome.

At ASH 2025, the VAYHIT3 trial revealed that ianalumab, a BAFF-targeting monoclonal antibody, reduced bleeding and depleted B cells in primary immune thrombocytopenia, offering hope for treatment-resistant patients. The FDA approved etuvetidigene autotemcel for Wiskott-Aldrich syndrome, marking a gene therapy milestone. Other highlights included bezuclastinib’s biomarker reduction in non-advanced systemic mastocytosis and cardiovascular benefits from GLP-1 agonists.