Rilzabrutinib is marketed as WAYRILZ, an oral BTK kinase inhibitor approved for adults with persistent or chronic immune thrombocytopenia after insufficient response to prior treatment. The prescribing information describes immune-modulatory activity through inhibition of B-cell activation and interruption of Fc gamma receptor-mediated phagocytosis. Initial U.S. approval was in 2025.
Novartis received EU approval for Rhapsido (remibrutinib) as the first oral targeted treatment for chronic spontaneous urticaria. Phase 3 data also showed significant efficacy in chronic inducible urticaria, with filings underway for additional indications.
Sanofi's oral BTK inhibitor rilzabrutinib received orphan drug designation in Japan for IgG4-related disease and warm autoimmune hemolytic anemia, while also securing U.S. FDA breakthrough therapy designation for the rare anemia indication.
Japan's Ministry of Health, Labour and Welfare has granted orphan drug designation to Sanofi's rilzabrutinib for IgG4-related disease, marking the third global orphan designation for this indication based on positive phase 2 study data.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07216079 |
Rilzabrutinib for the Adult Participants With Chronic ITP Who Have Completed Phase 3 Study in Japan |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT07190196 |
A 52-week Study of Rilzabrutinib Efficacy and Safety Compared to Placebo in Adults Diagnosed With IgG4-related Disease |
RECRUITING | PHASE3 |
| NCT07086976 |
A Study to Investigate the Efficacy, Safety, and Pharmacokinetics of Oral Rilzabrutinib Compared With Placebo in Participants 18 Years of Age and Older With Warm Autoimmune Hemolytic Anemia |
RECRUITING | PHASE3 |
| NCT07007962 |
Study to Evaluate the Efficacy and Safety of Oral Rilzabrutinib in Adults With Immune Thrombocytopenia (ITP) Who Failed First-line Treatment |
RECRUITING | PHASE3 |
| NCT06975865 |
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease |
RECRUITING | PHASE3 |
| NCT06500702 |
A Study to Evaluate the Efficacy and Safety of Frexalimab, Brivekimig, or Rilzabrutinib in Participants Aged 16 to 75 Years With Primary Focal Segmental Glomerulosclerosis or Minimal Change Disease |
RECRUITING | PHASE2 |
| NCT06444204 |
A Study to Evaluate the Effect of Mild and Moderate Hepatic Impairment on the Single-Dose Pharmacokinetics of Rilzabrutinib (PRN1008) |
COMPLETED | PHASE1 |
| NCT06444191 |
Pharmacokinetics (PK) of Rilzabrutinib (PRN1008) in Healthy Japanese and Caucasian Subjects |
COMPLETED | PHASE1 |
| NCT05107115 |
Rilzabrutinib for the Treatment of Chronic Spontaneous Urticaria in Patients Who Remain Symptomatic Despite the Use of H1 Antihistamine |
COMPLETED | PHASE2 |
| NCT05104892 |
Proof of Concept Study of Rilzabrutinib in Adult Participants With Moderate-to-severe Asthma |
COMPLETED | PHASE2 |
