Novartis’ Ianalumab Shows Promise in Phase 3 Trial for Rare Autoimmune Disorder
Novartis has announced positive results from a pivotal phase 3 trial investigating its experimental drug ianalumab in patients with primary immune thrombocytopenia (ITP), a rare autoimmune condition that causes the immune system to destroy platelets.
The disease, characterised by an increased risk of bleeding and fatigue, often proves difficult to manage when conventional corticosteroid treatments fail.
Strong Results from VAYHIT2 Trial
The late-stage study, known as VAYHIT2, evaluated ianalumab in combination with eltrombopag in patients who had not responded adequately to corticosteroids. Results revealed that patients treated with ianalumab were able to maintain safe platelet levels for longer periods compared to those receiving a placebo.
Importantly, ianalumab also delivered superior outcomes on the key secondary endpoint: at six months, patients receiving the drug demonstrated higher rates of sustained platelet count improvements.
Convenience of Once-Monthly Dosing
Ianalumab was administered as four once-monthly doses, a schedule that could potentially provide patients with extended periods of disease control without the burden of continuous treatment. Safety findings remained consistent with earlier studies, with no new concerns observed.
A professor of medicine at the University of Pennsylvania described the results as “encouraging,” noting that the findings suggest ianalumab may support longer disease control while reducing the need for ongoing therapy.
Next Steps and Regulatory Pathway
Novartis plans to present full data from the VAYHIT2 trial at an upcoming medical meeting. Regulatory submissions are expected in 2027, with data from this study set to be combined with findings from an ongoing first-line ITP trial.
Ianalumab has already received Orphan Drug Designation from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), reflecting its potential in addressing an area of significant unmet need.
Beyond ITP, the drug is also being explored in other autoimmune conditions such as Sjögren’s disease and lupus.
Conclusion
The VAYHIT2 results mark a promising step forward for Novartis in the treatment of primary immune thrombocytopenia. By demonstrating both improved platelet stability and the potential for less frequent dosing, ianalumab could transform care for patients with this challenging condition.
With regulatory submissions on the horizon and broader studies underway, the therapy may soon represent a new frontier in autoimmune disease management.
News Credits: Novartis blood disorder drug shows promise in late-stage trial
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