By VETTAPHARMA reporter – Derek Roche: Japan’s Ministry of Health, Labour and Welfare has approved Sanofi’s Wayrilz (rilzabrutinib) for the treatment of persistent or chronic immune thrombocytopenia (ITP) in patients who have responded inadequately to existing therapies or for whom tolerability has become problematic.
The approval introduces a novel oral Bruton’s tyrosine kinase (BTK) inhibitor that works through multi-immune modulation to address the underlying pathophysiology of ITP rather than solely increasing platelet counts.
The approval is based on results from the pivotal Phase 3 LUNA 3 trial (NCT04562766), which met both its primary and secondary endpoints. The study enrolled 202 adults with persistent or chronic ITP and demonstrated that Wayrilz significantly improved sustained platelet responses and other disease-related symptoms compared with placebo. Patients achieving a platelet response at 12 weeks continued into the full 24-week double-blind treatment period, including 64% of patients receiving Wayrilz versus 32% receiving placebo.
At Week 25, 23% of patients treated with Wayrilz achieved a durable platelet response, compared with 0% in the placebo group (p<0.0001). The therapy also produced a significantly faster time to first platelet response (36 days versus not reached with placebo; p<0.0001) and a longer duration of platelet response (7 weeks versus 0.7 weeks, least-square mean).
Patients receiving Wayrilz also reported greater improvements in quality of life. Scores on the Immune Thrombocytopenia Patient Assessment Questionnaire improved by 10.6 points in the overall quality-of-life domain compared with 2.3 points for placebo, although this analysis was descriptive and not powered for statistical significance. The most commonly reported adverse reactions were diarrhea, nausea, headache, abdominal pain, and COVID-19.
Wayrilz is a novel oral reversible covalent BTK inhibitor developed using TAILORED COVALENCY® technology to selectively inhibit BTK and restore immune balance through multi-immune modulation. According to Sanofi, the medicine is now approved for ITP in the United States, European Union, United Arab Emirates, United Kingdom, and Japan, while additional studies are ongoing in IgG4-related disease, warm autoimmune hemolytic anemia, and sickle cell disease.
Quick FAQs
1. What Is Wayrilz?
Wayrilz (rilzabrutinib) is a novel oral reversible Bruton’s tyrosine kinase (BTK) inhibitor that uses multi-immune modulation to address the underlying immune dysregulation responsible for immune thrombocytopenia.
2. What Happened?
Japan approved Sanofi’s Wayrilz for the treatment of persistent or chronic immune thrombocytopenia in patients who have not responded adequately to existing therapies or cannot tolerate them.
3. What Is The LUNA 3 Study?
LUNA 3 (NCT04562766) is a Phase 3, placebo-controlled study evaluating the efficacy and safety of Wayrilz in 202 adults with persistent or chronic immune thrombocytopenia.
4. What Is Immune Thrombocytopenia?
Immune thrombocytopenia is a rare immune-mediated disorder characterized by platelet counts below 100,000/µL, increasing the risk of bleeding, thromboembolism, fatigue, cognitive impairment, and reduced quality of life.
5. What Were The Key Results?
Wayrilz achieved a 23% durable platelet response versus 0% with placebo at Week 25, shortened the time to first platelet response to 36 days, prolonged platelet response duration, and improved patient-reported quality of life.
6. Why Is This Important?
The approval provides patients with persistent or chronic ITP in Japan with a new oral treatment that targets the underlying immune mechanisms of the disease, offering an additional therapeutic option for those with inadequate responses or poor tolerability to existing therapies.
