FDA

Wayrilz OK’d for persistent or chronic immune thrombocytopenia

September 5, 2025

Brand name: Wayrilz

Generic name: rilzabrutinib

Manufacturer: Sanofi

Approval date: August 29, 2025

FDA approved Wayrilz (rilzabrutinib), a small-molecule, reversible Bruton tyrosine kinase (BTK) inhibitor, for adults with persistent or chronic immune thrombocytopenia (ITP) who’ve had an insufficient response to immunoglobulins, anti-D therapy, or corticosteroids.

Efficacy

Wayrilz’s safety and efficacy was evaluated in the 24-week, double-blind, parallel-group LUNA-3 trial (NCT04562766) looking at durable platelet response (i.e., an increased number of platelets to ≥50 x 10⁹/L from baseline lasting the majority of the last 12 weeks of the treatment period). In this study, 202 patients were randomly assigned to either the Wayrilz group (133) or placebo group (69). During the 24-week treatment period, 31 (23%) patients in the Wayrilz group and no patients in placebo group achieved a sufficient and durable platelet count response.

Safety

Wayrilz may increase the risk of serious infections (including bacterial, viral, or fungal). The most common adverse reactions (≥10%) in the LUNA-3 trial were diarrhea, nausea, headache, abdominal pain, and COVID-19.

Recommended dose

The recommended dosage of Wayrilz is 400 mg taken orally twice daily. Patients who experience GI symptoms should consider taking Wayrilz with food to improve tolerability. Patients should swallow the pill whole with a glass of water; the pill should not be cut, crushed, or chewed.

Sources:

FDA Approves Drug to Treat Adults with Persistent or Chronic Immune Thrombocytopenia [News release]. 2025. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-treat-adults-persistent-or-chronic-immune-thrombocytopenia

Press release: Sanofi’s Wayrilz approved in US as first BTK inhibitor for immune thrombocytopenia [News release]. 2025. https://www.sanofi.com/en/media-room/press-releases/2025/2025-08-29-21-50-18-3141825

Wayrilz (rilzabrutinib) [package insert]. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219685s000lbl.pdf Revised August 2025. Accessed September 4, 2025.