Sanofi's Rilzabrutinib Shows Promise in Treating Rare Platelet Disorder

Sanofi's rilzabrutinib, an oral treatment for immune thrombocytopenia (ITP), showed promising results in a Phase 3 trial, improving platelet response and reducing bleeding episodes. The drug, a bruton's kinase inhibitor, is safer and better tolerated than existing treatments, potentially setting a new standard for ITP therapy. Meanwhile, Novo Nordisk's etavopivat reduced severe pain crises in sickle cell disease patients in a Phase 2 study, though results were not statistically significant. Beam Therapeutics' CRISPR-based therapy for sickle cell disease also showed consistent positive results, with no severe pain crises reported among treated patients.