Novartis' Ianalumab Exceeds Expectations in Phase 3 Trials for Primary Immune Thrombocytopenia Treatment

Novartis's ianalumab drug, combined with eltrombopag, has met the primary endpoint in a phase 3 trial for primary immune thrombocytopenia. The drug prolonged safe platelet levels during and after treatment, and patients experienced a significantly higher rate of sustained platelet count improvements. Novartis plans to present the data at a medical meeting and include it in future regulatory submissions in 2027.

Novartis has reported positive top-line results from the Phase 3 VAYHIT2 trial evaluating ianalumab in combination with eltrombopag for the treatment of primary immune thrombocytopenia (ITP). The trial met its primary endpoint, demonstrating that ianalumab prolonged safe platelet levels during and after treatment. Patients treated with ianalumab experienced a significantly higher rate of sustained improvements in platelet count, the key secondary endpoint of the study [1].

The trial involved 300 patients with ITP who had previously failed first-line treatment with corticosteroids. Participants were randomized to receive either ianalumab at 3 mg/kg or 9 mg/kg, or placebo, in addition to eltrombopag. The primary endpoint was time to treatment failure, defined as the time from randomization until either a platelet count of less than 30 G/L later than 8 weeks from randomization, the need for rescue therapy later than 8 weeks from randomization, initiation of a new ITP treatment at any time, or death [2].

The safety profile of ianalumab was consistent with previous clinical studies, with no new safety signals observed. The 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 [1].

ITP is a rare autoimmune disorder characterized by low platelet counts, leading to an increased risk of bleeding, bruising, and chronic fatigue. Despite available treatments, many patients cycle through multiple therapies, unable to achieve long-term disease control [3]. The positive results from VAYHIT2 suggest that ianalumab may offer longer periods of disease control and reduce the need for continuous treatment [1].

The trial results highlight the potential of ianalumab to provide durable responses in ITP patients, potentially reducing the burden of long-term treatment. If approved, ianalumab could offer a significant advancement in the treatment of ITP, providing patients with extended time off treatment and improved quality of life [1].

References:

[1] https://finance.yahoo.com/news/novartis-ianalumab-phase-iii-trial-051500851.html

[2] Clinicaltrials.gov. NCT05653219. A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids (VAYHIT2). Accessed July 21, 2025. https://clinicaltrials.gov/study/NCT05653219

[3] Rodeghiero F, Stasi R, Gernsheimer T,