Twenty-three percent of patients treated with rilzabrutinib achieved a durable platelet response at week 25 compared with none in the placebo group.

Approval was based on results from the phase 3 LUNA 3 trial that was presented at the 66th American Society of Hematology Annual Meeting and Exposition, which evaluated efficacy and safety in 202 patients.

Rilzabrutinib is an oral, reversible Bruton’s tyrosine kinase inhibitor designed to modulate multiple immune pathways implicated in immune thrombocytopenia pathophysiology. Median time to first platelet response was 36 days with rilzabrutinib versus not reached with placebo (p<0.0001). Mean duration of platelet response was 7 weeks for the active arm versus 0.7 weeks for placebo.

Secondary measures demonstrated improvement in patient-reported outcomes. Participants receiving rilzabrutinib reported a 10.6-point improvement across 9 quality-of-life domains compared with a 2.3-point increase with placebo, although these analyses were not powered for statistical significance. The most common adverse events (≥10%) included diarrhea, nausea, headache, abdominal pain, and COVID-19.

Immune thrombocytopenia is characterized by complex immune dysregulation that results in low platelet counts, bleeding symptoms, and reduced quality of life. Rilzabrutinib represents a novel approach by using multi-immune modulation to help address the root causes of immune thrombocytopenia.

“Through multi-immune modulation, Wayrilz can offer a new option for patients, including those who fail steroids or do not respond to existing treatment.” said LUNA 3 study author David Kuter, MD, Director of Clinical Hematology at Massachusetts General Hospital, Professor of Medicine at Harvard Medical School. 

Rilzabrutinib was previously approved in the United Arab Emirates and is under regulatory review in the European Union and China. It has received orphan drug designations in multiple regions for immune thrombocytopenia and additional rare immune-mediated conditions.

Source: Sanofi