Novartis’ ianalumab has shown success in the third trial readout in two days, after it met both its endpoints in a Phase III immune thrombocytopenia (ITP) trial.
During the VAYHIT2 trial (NCT05653219), a combination of the monoclonal antibody (mAb) plus Promacta (eltrombopag) significantly prolonged the time to treatment failure (TTF) – meeting its primary endpoint for TTF improvements versus Promacta and placebo.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
TTF was classified as the duration in which a patient could maintain safe platelet levels during and after treatment.
The four-time, once-monthly therapy also met its secondary endpoint – offering a sustained and significant increase in platelet counts after six months of treatment.
These trends were seen in patients who had previously been treated with corticosteroids, the first-line standard of care (SoC) for this condition.
Top-line results further validated the safety profile of ianalumab, with no new signals being observed within this trial.
US Tariffs are shifting - will you react or anticipate?
Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.
By GlobalDataNovartis said it hopes to file with global regulators for approval of the drug in ITP in 2027, with supporting evidence from the ongoing first-line ITP trial, VAYHIT1 (NCT05653349).
If approved in ITP, it would reduce patient reliance on continuous treatment – offering patients more time between hospital trips and boosting quality of life.
This could significantly impact the disease’s treatment landscape, which has typically relied on regular hospital-administered intravenous treatments like immunoglobulin infusions and rituximab when corticosteroids fail.
In the 12 August announcement, Novartis CMO Shreeram Aradhye “For many living with ITP, chronic treatment is disruptive to their daily life due to the burden of regular dosing, dose adjustments and side effects.
“The positive top-line results of the VAYHIT2 trial highlight the potential of ianalumab, if approved, to deliver long-term disease control, while enabling extended time off treatment.”
This latest success comes just 24 hours after the drug’s success in the Phase III NEPTUNUS-1(NCT05350072) and NEPTUNUS-2 (NCT05349214) trials, which put it in the running as the first targeted therapy to gain approval for Sjögren’s disease, released on 11 August.
Stiff competition ahead for ianalumab
Though the top-line results for ianalumab in ITP look promising for Novartis, the Swiss pharma may face significant competition from Sanofi’s Bruton’s tyrosine kinase inhibitor (TKI) rilzabrutinib, which is currently in the pre-registration phase for the indication.
Since the drug is administered orally, it could offer patients a more convenient treatment option that can be taken independently if approved – potentially eclipsing ianalumab’s benefits over other regular intravenous interventions.
Sanofi’s drug is currently under regulatory review in the US, EU and China, with the FDA expected to make a decision on the drug by 29 August 2025.
GlobalData’s analysts forecast that rilzabrutinib and ianalumab will be neck-and-neck in the indication, with the former estimated to pull in $607m by 2031, while the latter will likely make $638m by the same year.
GlobalData is the parent company of Clinical Trials Arena.
Since the ITP market as a whole was worth $1.1bn in 2024 – less than these two drugs alone – these figures suggest that the indication is poised for strong growth moving forward.
