EMA highlighted that rilzabrutinib significantly improves durable platelet response, especially when combined with corticosteroids or thrombopoietin receptor agonists, based on phase 3 clinical trial data.

ITP is a rare autoimmune disorder characterized by low platelet counts caused by autoantibody-mediated destruction and impaired production, which increases bleeding risk.

The European Medicines Agency (EMA) has recommended marketing authorization for rilzabrutinib (Wayrilz, Sanofi) as a treatment for immune thrombocytopenia (ITP) in adults who do not respond to other therapies.

Common side effects of rilzabrutinib include diarrhea, nausea, headache, increased risk of infections, abdominal pain, joint pain, and nasopharyngitis, with potential hepatotoxicity and drug interactions.

ITP can develop after viral infections, vaccinations, or certain medications, and while most cases resolve in children, over half of adult cases become chronic, increasing the risk of severe bleeding.

First-line treatments like corticosteroids often fail, with up to 75% of patients relapsing within three to four months, leading to higher healthcare costs due to additional therapies and hospitalizations.

The incidence of ITP varies across Europe, affecting about 5 per 100,000 children and 2 per 100,000 adults annually, with symptoms ranging from mild to severe bleeding, including rare fatal intracranial hemorrhages.

Prescribers are advised to administer rilzabrutinib under the supervision of experienced hematologists, avoiding co-administration with CYP3A inhibitors or inducers and proton pump inhibitors.

Rilzabrutinib is a first-in-class Bruton’s tyrosine kinase inhibitor that modulates the immune response by inhibiting B cell activation, FcγR-mediated phagocytosis, and reducing chronic inflammation associated with ITP.