Overview
- The EMA’s CHMP issued a positive opinion on 16 October 2025 recommending marketing authorisation for Wayrilz (rilzabrutinib) to treat adult immune thrombocytopenia patients who are refractory to other therapies.
- The recommendation is supported by a phase 3 multicentre, randomised, double-blind, placebo-controlled trial showing significantly higher durable platelet responses versus placebo, particularly alongside corticosteroids or thrombopoietin receptor agonists.
- Rilzabrutinib is a Bruton’s tyrosine kinase inhibitor that modulates immune activity by inhibiting B cell activation and interrupting FcγR‑mediated phagocytosis.
- Common adverse reactions reported include diarrhoea, nausea, headache, COVID-19, abdominal pain, arthralgia and nasopharyngitis.
- Wayrilz will be supplied as 400 mg film-coated tablets by applicant Sanofi B. V., and if authorised the EMA will publish the SmPC and review maintenance of the product’s orphan designation.