The US FDA approves Sanofi’s Wayrilz (rilzabrutinib) for use in adult patients with chronic or persistent immune thrombocytopenia (ITP) who did not respond adequately to prior therapies. The FDA approves Sanofi’s Wayrilz after the LUNA 3 phase 3 pivotal trial, during which the drug achieved its primary and secondary endpoints, showing favorable impacts on sustained platelet count and the alleviation of ITP-related complications.
“The burden of immune thrombocytopenia can be both physical and emotional with significant overlooked symptoms that can impact various aspects of daily living,” said Caroline Kruse, President and CEO at the Platelet Disorder Support Association. “We are pleased to have a new treatment option that can help ease the ongoing strain of managing the disease for patients and their families.”
Wayrilz is an oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor designed to act on the root cause of ITP. Its multi-immune modulation mechanism targets several pathways across the immune system.
The LUNA 3 phase 3 study, presented at the 66th American Society of Hematology Annual Meeting and Exposition, assessed the efficacy and safety of Wayrilz compared with placebo in adults with persistent or chronic ITP. Patients who reached platelet response at week 12 were permitted to complete the 24-week double-blind stage. In total, 64% of patients in the Wayrilz group and 32% in the placebo group met this milestone. The study showed:
- A statistically significant durable platelet response at week 25 (23% of Wayrilz patients vs. 0% with placebo; p<0.0001).
- Quicker time to first platelet response (36 days with Wayrilz vs. not reached with placebo; p<0.0001).
- A longer platelet response duration (7 weeks with Wayrilz vs. 0.7 weeks with placebo).
Beyond platelet improvement, patients in the Wayrilz arm reported a 10.6-point increase across nine quality-of-life measures, compared with a 2.3-point gain in the placebo group. This result was assessed with the Immune Thrombocytopenia Patient Assessment Questionnaire, which is intended to record actual patient experience of ITP symptoms.
The most common adverse reactions seen in greater than 10% of patients were diarrhea, nausea, headache, abdominal pain, and COVID-19.
“Traditionally, immune thrombocytopenia management has focused on restoring platelet counts and reducing bleeding risk, which for some patients may result in suboptimal responses, persistent symptoms, or unacceptable treatment complications,” said David Kuter, MD, Director of Clinical Hematology at Massachusetts General Hospital and Professor of Medicine at Harvard Medical School, and study author. “Through multi-immune modulation, Wayrilz can offer a new option for patients, including those who fail steroids or do not respond to existing treatment.”
Earlier this year, Wayrilz received approval in the United Arab Emirates for adults with ITP who had inadequate response or intolerance to prior treatment. Regulatory reviews are also ongoing in the European Union and China.
The therapy previously received Fast Track and Orphan Drug Designations for ITP in the US, as well as orphan designations in Japan and the EU. More recently, the FDA expanded its Orphan Drug status to include three additional rare diseases: warm autoimmune hemolytic anemia (wAIHA), IgG4-related disease (IgG4-RD), and sickle cell disease (SCD). Wayrilz has also been granted Fast Track Designation by the FDA and orphan designation by the European Medicines Agency in IgG4-RD.
