European Commission grants approval to Sanofi's Wayrilz for refractory immune thrombocytopenia | iPharmaCenter

European Commission grants approval to Sanofi's Wayrilz (rilzabrutinib), marking the first Bruton's tyrosine kinase (BTK) inhibitor for adult patients with refractory immune thrombocytopenia (ITP).

Wayrilz, an oral and reversible BTK inhibitor, targets immune pathways through multi-immune modulation to tackle ITP's root causes, such as low platelet counts, bleeding risks, and quality-of-life burdens from this rare autoimmune disorder. The approval follows a positive CHMP opinion and supports use in adults unresponsive to prior therapies.

LUNA 3 Study Results

The pivotal LUNA 3 phase 3 trial (NCT04562766) enrolled 202 adults with persistent or chronic ITP, comparing Wayrilz 400 mg twice daily to placebo over a 24-week double-blind period. Key outcomes included a 23% durable platelet response rate at week 25 (vs. 0% placebo), faster first response (36 days vs. not reached), and longer response duration (7 weeks vs. 0.7 weeks). Quality-of-life scores improved by 10.6 points on Wayrilz versus 2.3 points on placebo per the ITP Patient Assessment Questionnaire, though not statistically powered.

Study Design Highlights

This multicenter trial featured a primary endpoint of platelet counts ≥50,000/μL for at least 8 of the last 12 weeks without rescue therapy. Secondary measures covered response time, duration, rescue use, fatigue, and bleeding via the ITP Bleeding Scale at week 25, with an ongoing adolescent cohort. Results were presented at the 66th ASH meeting and published in Blood.

Global Status and Mechanism

Wayrilz is approved for ITP in the US, EU, and UAE, with reviews ongoing in Japan and China; it holds orphan and fast-track designations in multiple regions. Sanofi's TAILORED COVALENCY enables selective BTK inhibition in B cells and macrophages, addressing immune dysregulation. Common side effects include diarrhea, nausea, headache, and abdominal pain