Sanofi’s rilzabrutinib demonstrates significant benefit in phase 3 study of a BTK inhibitor in ITP

Sanofi releases their new positive results from the pivotal LUNA 3 phase 3 study of rilzabrutinib in adults with persistent or chronic immune thrombocytopenia, a rare immune-mediated disease, reinforce the efficacy and safety of rilzabrutinib, an oral, reversible, covalent Bruton’s tyrosine kinase inhibitor, and further support its potential as a first-in-class treatment for ITP. Platelet response was achieved in 65% of patients receiving rilzabrutinib compared to 33% of patients on placebo.

David Kuter, MD
Director of Clinical Hematology at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School, study author
“People living with immune thrombocytopenia who cannot tolerate or do not respond to medications aimed at raising platelet counts are at risk of uncontrolled bleeding and often endure side effects from steroids and other available therapies. A significant percentage of these patients also suffer from severe fatigue and an impaired quality of life. I’m encouraged by the robust therapeutic effects I’ve seen in patients of the LUNA 3 study across all aspects of the disease, including clinically meaningful and sustained improvements in platelet count, quality of life metrics, reduction in bleeding, and a favorable safety profile.”

The safety profile of rilzabrutinib was consistent with previous studies. The rates of adverse events (AEs) were similar in patients receiving rilzabrutinib and patients receiving placebo; the most common treatment-related AEs for rilzabrutinib were mild/moderate (grade 1/2), including diarrhea (23%), nausea (17%), headache (8%) and abdominal pain (6%).

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positive phase 3 study of a BTK inhibitor in ITP, rilzabrutinib, Sanofi