The Ministry of Health, Labour and Welfare (MHLW) in Japan has granted marketing and manufacturing authorisation to Wayrilz (rilzabrutinib), a novel oral reversible Bruton’s Tyrosine Kinase inhibitor (BTKi), for the treatment of persistent or chronic immune thrombocytopenia (ITP) in patients who do not respond sufficiently to other treatments or in whom tolerability is considered to be problematic.

Wayrilz can help address the underlying causes of the disease, through multi-immune modulation, targeting key pathways across the immune system.

The approval is based on the LUNA 3 phase 3 study (clinical study identifier: NCT04562766), in which Wayrilz met the primary and secondary endpoints, showing a positive impact on sustained platelet counts and other symptoms.

Hisashi Kato, MD, PhD, Department of Blood Transfusion and Cell Therapy, The University of Osaka Hospital, Suita, Japan, said, "For ITP patients, achieving platelet counts close to normal and living without worrying about bleeding is extremely important. Additionally, in recent years, there has been growing interest in various other symptoms associated with ITP such as fatigue. Wayrilz is a novel BTKi that works through multi-immune modulation to help address the root cause of ITP pathophysiology. It is expected to become a new therapeutic option that may help patients with disease management, including not only improvement in platelet counts but also considering patients' quality of life."

The LUNA 3 phase 3 study evaluated the efficacy and safety of Wayrilz compared to placebo in adults (n=202) with persistent or chronic ITP. Patients who achieved platelet count response at 12 weeks were eligible to continue the full 24-week double-blind period (64 percent of patients in the Wayrilz arm and 32 percent of patients in the placebo arm).

Manuela Buxo, Executive Vice President (EVP), Global Head of Specialty Care, Sanofi, said, “People living with immune thrombocytopenia who continue to experience symptoms, despite trying existing treatments, face a great unmet need. Wayrilz has a differentiated mechanism of action that enables multi-immune modulation to address the underlying pathophysiology of ITP and offers new hope to patients who have not responded to prior therapy. This milestone underscores Sanofi’s commitment to bringing innovative treatment options to rare disease patients around the world.”

Compared to patients receiving placebo, patients receiving Wayrilz experienced statistically significant durable platelet response at week 25 (23 percent of patients in Wayrilz arm vs. zero percent in placebo arm; p<0.0001); faster time to first platelet response (36 days in Wayrilz arm vs. not reached in placebo arm; p<0.0001); and longer duration of platelet response (least square mean of 7 weeks in Wayrilz arm vs. 0.7 weeks in placebo arm).

Patients receiving Wayrilz reported an overall 10.6-point improvement in the overall quality of life domain compared to a 2.3-point increase in the placebo arm, based on The Immune Thrombocytopenia Patient Assessment Questionnaire, a clinical tool designed to measure ITP symptoms and impacts. The results of this analysis are descriptive and were not powered for statistical significance.

The most common adverse reactions (incidence ≥10 percent) were diarrhea, nausea, headache, abdominal pain, and COVID-19.

Wayrilz is being studied across multiple other rare diseases, including IgG4-Related Disease (IgG4-RD), warm autoimmune hemolytic anemia (wAIHA), and sickle cell disease. These additional indications are currently under investigation and have not been approved by regulatory authorities. Japan has granted Wayrilz Orphan Drug Designation (ODD) for ITP, IgG4-RD, and wAIHA.