ASH: rilzabrutinib demonstrated significant patient benefit in the first positive phase 3 study of a BTK inhibitor in ITP
Pivotal phase 3 data show rapid and durable platelet response, reduced bleeding and need for rescue response, and improved physical fatigue and quality of life measures in patients with persistent or chronic ITPResults underscore the safety and efficacy of rilzabrutinib and its potential as the first BTK inhibitor in ITPRilzabrutinib is currently under regulatory review in the US and the EU
Paris, December 7, 2024. Positive results from the pivotal LUNA 3 phase 3 study of rilzabrutinib in adults with persistent or chronic immune thrombocytopenia (ITP), a rare immune-mediated disease, reinforce the efficacy and safety of rilzabrutinib, an oral, reversible, covalent Bruton’s tyrosine kinase (BTK) inhibitor, and further support its potential as a first-in-class treatment for ITP. Platelet response was achieved in 65% (n=86) of patients receiving rilzabrutinib compared to 33% (n=23) of patients on placebo. The primary endpoint was met, with rilzabrutinib demonstrating durable platelet response in 23% of ITP adult patients compared to 0% on the placebo arm (p
