San Diego, CA – Phase 3 data from the LUNA 3 trial support rilzabrutinib (Sanofi) as a potential first-in-class oral Bruton tyrosine kinase (BTK) inhibitor for patients with previously treated immune thrombocytopenia (ITP). The treatment showed rapid and durable platelet responses, reduced bleeding, and improved health-related quality of life in patients.
Rilzabrutinib achieved significant improvements in platelet counts, with nearly two-thirds of patients showing a response at week 13. Patients taking rilzabrutinib were three times more likely to achieve a platelet response than those on placebo. The treatment also reduced the need for rescue therapy by 52% and showed sustained improvement in physical fatigue.
While the most common adverse events with rilzabrutinib were mild to moderate, the study found a favorable safety profile overall. A total of one patient experienced a grade 3 peripheral embolism and another died due to pneumonia unrelated to treatment.
The LUNA 3 trial demonstrated significant progress in treating ITP, a relatively rare autoimmune disease that affects 10-23 patients per 100,000 in the US. With rilzabrutinib, Sanofi is one step closer to bringing a new treatment option for this patient population.
Rilzabrutinib received fast-track designation from the FDA in November 2020 and is currently under regulatory review with a target action date of August 29, 2025. The study’s findings are promising, but further research is needed to confirm its efficacy and safety.
Source: https://www.medscape.com/viewarticle/rilzabrutinib-shines-phase-3-trial-tough-treat-immune-2024a1000mlv